Two high-tech companies have teamed up to take 3D printing in space to the next level. A new 3D printer, sent to the space station on a SpaceX cargo resupply mission last July, is now officially open for business. Its goal: to print human tissue in space.

Formally known as the 3D BioFabrication Facility (or BFF), the printer will use adult human cells (like stem cells) as its feedstock. The BFF is just the first step to a much larger goal of printing human organs such as hearts or lungs in space.

The initial phase for BFF, which could last about two years, will involve creating test prints of cardiac-like tissue of increasing thickness, Techshot representatives said in a statement. (Techshot is collaborating on the project with nScrypt, a 3D bioprinter and electronic printer manufacturer.)

If all goes according to plan, the company would then graduate to printing heart patches in space. Once printed, they would be shipped back to Earth and tested in small animals (such as rats) to see how they do. The next step after that could be entire organs.

Ultimately, long-term success of BFF could lead to reducing the current shortage of donor organs and eliminate the requirement that someone must first die in order for another person to receive a new heart, other organ or tissue, Techshot said.

Imagine needing a organ and instead of having to wait on the transplant list for an one that could never come, using a bit of your own DNA, a new organ could be printed for you in space.

Researchers on Earth have celebrated some success with the 3D printing of bones and cartilage, but when it comes to soft tissues, they havent had the same luck.

Tissues collapse under their own weight due to gravity. This results in not much more than a puddle of biomaterial. But when these sames components are used in space, they retain their shape.

However, without additional conditioning, once these new tissues return to Earth, theyd collapse just like there terrestrial counterparts. Heres where BFF comes in.

In addition to launching a bioprinter, Techshot has also developed a means of curing the newly printed tissues. This way they will remain solid even after returning to Earth. The company says that the actual printing process will take less than a day, the strengthening process will take an estimated 12-45 days. It all depends on the tissue.

This could lead to less people dying as they wait on transplant lists, and it could also mean less dependency on anti-rejection medications. Assembling a whole human organ (such as a heart or lung) was once strictly science fiction. While its still a few years away, it is now a possibility.

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3D printed organs are coming to an International Space Station near you - Teslarati

Cardiac Stem Cells Comments Off on 3D printed organs are coming to an International Space Station near you – Teslarati | January 30th, 2020

ANN ARBORConventional thinking is that bone regeneration is left to a small number of mighty cells called skeletal stem cells, which reside within larger groups of bone marrow stromal cells.

But new findings from the University of Michigan recasts that thinking.

In a recent study, Noriaki Ono, assistant professor at the U-M School of Dentistry, and colleagues report that mature bone marrow stromal cells metamorphosed to perform in ways similar to their bone-healing stem cell cousinsbut only after an injury.

Bone fracture is an emergency for humans and all vertebrates, so the sooner cells start the business of healing damaged boneand the more cells there are to do itthe better.

Our study shows that other cells besides skeletal stem cells can do this job as well, Ono said.

In the mouse study, inert Cxcl12 cells in bone marrow responded to post-injury cellular cues by converting into regenerative cells, much like skeletal stem cells. Normally, the main job of these Cxcl12-expressing cells, widely known as CAR cells, is to secrete cytokines, which help regulate neighboring blood cells. They were recruited for healing only after an injury.

The surprise in our study is that these cells essentially did nothing in terms of making bones, when bones grow longer, Ono said. Its only when bones are injured that these cells start rushing to repair the defect.

This is important because the remarkable regenerative potential of bones is generally attributed to rare skeletal stem cells, Ono says. These new findings raise the possibility that these mighty skeletal stem cells could be generated through the transformation of the more available mature stromal cells.

These mature stromal cells are malleable and readily available throughout life, and could potentially provide an excellent cellular source for bone and tissue regeneration, Ono says.

The study appears in the journal Nature Communications.

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After a bone injury, shape-shifting cells rush to the rescue - University of Michigan News

Bone Marrow Stem Cells Comments Off on After a bone injury, shape-shifting cells rush to the rescue – University of Michigan News | January 30th, 2020

A dad who saved the life of a cancer patient 6,600 miles away was flown around the world on a trip of a lifetime by his grateful recipient - who tracked him down.

Milton Becker, 69, was close to death and in desperate need of a bone marrow donor when a two-and-a-half year global search linked him with an anonymous Welsh man.

Emyr Williams, 54, was a near-perfect match, and his bone marrow was flown to Canada and given to Milton, who was declared cancer free.

The pair were linked up by the donation database and grew close via phone calls and Facebook messages.

And last year he invited retired carpenter Emyr to Canada for a two week body">

A dad who saved the life of a cancer patient 6,600 miles away was flown around the world on a trip of a lifetime by his grateful recipient - who tracked him down.

Milton Becker, 69, was close to death and in desperate need of a bone marrow donor when a two-and-a-half year global search linked him with an anonymous Welsh man.

Emyr Williams, 54, was a near-perfect match, and his bone marrow was flown to Canada and given to Milton, who was declared cancer free.

The pair were linked up by the donation database and grew close via phone calls and Facebook messages.

And last year he invited retired carpenter Emyr to Canada for a two week $15,000 (8,835) trip around Alberta and the Rocky Mountains.

Meeting him for the first time at the airport, wearing a Welsh dragon T-shirt and a Wales flag, they formed an instant bond.

Milton said he's "indebted" to his hero - and is planning a UK trip.

Dad-of-three Emyr, from Lampeter, Wales, said: "It was surreal to be out there.

"There's this bond between us like no other.

"It was only when we went out there that we really understood how close to death Milton was.

"One of his friends said he had been finalising plans to be at his funeral.

"He was literally on death's door.

"For something that required no real effort at all saved that great man's life.

"And to have the pleasure of meeting him in the flesh and to be introduced to his family was just an honour."

Granddad-of-two Milton, from Alberta, Canada, added: "We got on so well and I just thought I've got to thank this guy.

"I didn't want him to spend a penny. It was my treat.

"It's not about the money. What he did was priceless.

"I'm forever indebted to the guy."

Milton was diagnosed with stage 4 leukaemia in 2010 but after unsuccessful chemotherapy he was told a bone marrow transplant was the only means of survival.

Doctors searched across Canada but were unsuccessful and begun their two-and-a-half year worldwide search for a donor.

In early 2013, Emyr - who had been registered on the blood transfusion register for several years - was found to be a near-perfect match.

Emyr said: "A lady called me one day to say 'would you be interested in donating your stem cells?

"She went on to say there was a guy in Canada with leukaemia and that I was a 99.9999 per cent match with him.

"I just thought why not.

"It doesn't cost me anything and it can really change somebody's life."

The bone marrow was flown from Wales - with Milton receiving his long-awaited transfusion on his 63rd birthday, on 1st February 2013.

Former oil company lorry driver Milton said: "What he did was completely priceless.

"There's no better gift than the gift of life.

"And to get that on my birthday, well, it was a great feeling!"

A year after the transfusion Milton was told he was on the road to recovery but was kept in remission and monitored by doctors for the next two years.

In 2016, three years after the blood transfusion, Milton was deemed cancer-free.

It led nurses to ask Milton if he would like to know who his donor was - which he accepted straight away.

They got in touch with Emyr - who'd been given bi-annual anonymous updates - who agreed his details could be passed on.

Emyr said: "A few days later I had this call from an international number.

"I remember it as clear as day.

"He phoned me up and said; 'Emyr, my name is Milton and I just want to say how thankful I am'.

"From then on we just hit it off.

"What makes me laugh is he always forgets his Facebook password so he's a complete technophobe.

"We speak through his children on Facebook.

"We mostly speak about our family."

Milton said: "I couldn't turn up the chance to thank the guy who gave me life!

"I started off by thanking him and we had a great chat.

"I told him I would be forever grateful and wanted to keep in touch."

The two then added each other on Facebook and soon became good friends with weekly messages and monthly phone calls.

Then two years later Milton phoned Emyr to ask if he and his family would be interested in flying out to Canada for a two-week holiday.

Emyr said: "He asked me during one of our phone calls.

"I had never been to Canada and thought it would just be great to meet each other face-to-face."

Emyr flew out with his wife and teenage daughter last September 2019 to start the two-week itinerary around Alberta and the Rocky Mountains.

Emyr said: "He was there at the airport with a Welsh dragon on his T-shirt and a Welsh flag.

"You couldn't miss them.

"We have beautiful mountains here in Wales but Canada was just something else.

"It was an absolutely incredible trip.

"He paid for it all.

"We stayed in cabins, had a party with his extended family, we drank, sat by the open fire, and toasted marshmallows."

Milton added: "One Sunday I took him to my church.

"People knew he was coming and the service and to my surprise Emyr got up and told the church about the successful operation.

"There were tears but it was just beautiful."

Now seven years on from the transfusion, the pair say they are thankful to have one another in each other's lives.

The pair still keep regular contact with one another, with Facebook messages, fortnightly phone calls and even FaceTimed each other on Christmas Day.

Emyr said: "They're planning on coming to Wales next year in June or July.

"We'll definitely go back out there again in a few years.

"Even though we're thousands of miles away, we're such great friends."

Milton said: "We still have our chit-chats and I'd love to go over to the UK.

5,000 (8,835) trip around Alberta and the Rocky Mountains.

Meeting him for the first time at the airport, wearing a Welsh dragon T-shirt and a Wales flag, they formed an instant bond.

Milton said he's "indebted" to his hero - and is planning a UK trip.

Dad-of-three Emyr, from Lampeter, Wales, said: "It was surreal to be out there.

"There's this bond between us like no other.

"It was only when we went out there that we really understood how close to death Milton was.

"One of his friends said he had been finalising plans to be at his funeral.

"He was literally on death's door.

"For something that required no real effort at all saved that great man's life.

"And to have the pleasure of meeting him in the flesh and to be introduced to his family was just an honour."

Granddad-of-two Milton, from Alberta, Canada, added: "We got on so well and I just thought I've got to thank this guy.

"I didn't want him to spend a penny. It was my treat.

"It's not about the money. What he did was priceless.

"I'm forever indebted to the guy."

Milton was diagnosed with stage 4 leukaemia in 2010 but after unsuccessful chemotherapy he was told a bone marrow transplant was the only means of survival.

Doctors searched across Canada but were unsuccessful and begun their two-and-a-half year worldwide search for a donor.

In early 2013, Emyr - who had been registered on the blood transfusion register for several years - was found to be a near-perfect match.

Emyr said: "A lady called me one day to say 'would you be interested in donating your stem cells?

"She went on to say there was a guy in Canada with leukaemia and that I was a 99.9999 per cent match with him.

"I just thought why not.

"It doesn't cost me anything and it can really change somebody's life."

The bone marrow was flown from Wales - with Milton receiving his long-awaited transfusion on his 63rd birthday, on 1st February 2013.

Former oil company lorry driver Milton said: "What he did was completely priceless.

"There's no better gift than the gift of life.

"And to get that on my birthday, well, it was a great feeling!"

A year after the transfusion Milton was told he was on the road to recovery but was kept in remission and monitored by doctors for the next two years.

In 2016, three years after the blood transfusion, Milton was deemed cancer-free.

It led nurses to ask Milton if he would like to know who his donor was - which he accepted straight away.

They got in touch with Emyr - who'd been given bi-annual anonymous updates - who agreed his details could be passed on.

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Cancer patient flies dad who saved his life 6600 miles away around the world - Birmingham Live

Bone Marrow Stem Cells Comments Off on Cancer patient flies dad who saved his life 6600 miles away around the world – Birmingham Live | January 30th, 2020

A team based at Osaka University stated how it had succeeded in carrying out the first transplant of cardiac muscle cells, around the globe, developed from iPS cells in a clinical trial which as physician-initiated.

A professor in Osaka Universitys cardiovascular surgery unit, Yoshiki Sawa, along with his colleagues at the university, intend to transplant heart muscle cell sheets into 10 individuals experiencing severe heart malfunction as a result of ischemic cardiomyopathy, in a clinical trial, to validate the safety and the effectiveness of the therapy with the use of induced pluripotent stem cells.

On the surface of the hearts of the partaking individuals, the cells on the degradable sheets are attached. It is predicted that these cells will develop to release a protein that can allow for the regeneration of blood vessels as well as the improvement of the cardiac function.

Already, the iPS cells have been taken, and then stored, from the blood cells donated by healthy individuals

On Monday, the researchers stated how they chose to carry out a clinical trial in a clinical researchs stead as they had hoped to attain, as early as possible, authorization from the health ministry for clinical applications.

There are severe evaluating risks involved in the clinical trial. These may include the possibility of cancer as well as the efficacy of transplanting many million cells per patient, which may consist of tumor cells.

In Japan, this will be marked as the second clinical trial based on iPS. The first clinical trial of such kind was carried out on patients suffering from eye-linked ailments. This was done so by the Riken research institute.

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Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe - Medical Herald

IPS Cell Therapy Comments Off on Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe – Medical Herald | January 30th, 2020

GIOSTAR/HEAMGEN has developed and secured patented technology to manufacture lifesaving mature red blood cells from stem cells. The red blood cells are made utilizing a bioreactor that permits the production of mature red blood cells, under strictly controlled conditions, for transfusion therapy and replaces the need for a human blood donor. GIOSTAR/HEAMGEN mature red blood cells are safe and not compromised by inadequate pathogen detection and inactivation of diseases such as hepatitis C, HIV, hepatitis B and syphilis. The red blood cells are O-Negative (Universal Donor) to eliminate incompatibility and allosensitization reactions.

ATLANTA (PRWEB) January 29, 2020

GIOSTAR/HEAMGEN has developed and secured patented technology to manufacture lifesaving mature red blood cells from stem cells. The red blood cells are made utilizing a bioreactor that permits the production of mature red blood cells, under strictly controlled conditions, for transfusion therapy and replaces the need for a human blood donor. GIOSTAR/HEAMGEN mature red blood cells are safe and not compromised by inadequate pathogen detection and inactivation of diseases such as hepatitis C, HIV, hepatitis B and syphilis. The red blood cells are O-Negative (Universal Donor) to eliminate incompatibility and allosensitization reactions. Trauma situations often do not allow for adequate blood typing due to time restrictions, so the GIOSTAR/HEAMGEN red blood cells address that need effectively.

"There are three main problems for blood transfusions," stated Dr. Anand Srivastava, Founder and Chairman of GIOSTAR. "First we have to match the blood type. Second, there's not enough blood available every single time. And third, when we transfer blood from one person to another person, there is always a chance of the transfer of disease."

Watch a feature interview with Dr. Anand Srivastava on The DM Zone with host Dianemarie Collins.

The World Health Organization (WHO) published the first detailed analysis on the global supply and demand for blood in October 2019 and found that 119 out of 195 countries do NOT have enough blood in their blood banks to meet hospital needs. In those nations, which include every country in central, eastern, and western sub-Saharan Africa, Oceania (not including Australasia), and south Asia are missing roughly 102,359,632 units of blood, according to World Health Organization (WHO) goals. While total blood supply around the world was estimated to be around 272 million units, in 2017, demand reached 303 million units. That means the world was lacking 30 million units of blood, and in the 119 countries with insufficient supply, that shortfall reached 100 million units.

The global market opportunity for GIOSTAR/HEAMGEN technology presents not only a profitable and scalable business opportunity but also a significant social and environmental impact. The global market is estimated to be at least $ 85 Billion/year.

GIOSTAR/HEAMGEN has identified early entry global markets to include Military, Trauma, Asia (replace Hepatitis C contaminated blood products), Africa (AIDS contaminated blood), Newborns, Thalassemia patients, Allosensitized sickle cell disease patients. South Sudan was found to have the lowest supply of blood, at 46 units per 100,000 people. In fact, the country's need for blood was deemed 75 times greater than its supply. In India, which had the largest absolute shortage, there was a shortfall of nearly 41 million units, with demand outstripping supply by over 400 percent. Strategic investments are needed in many low-income and middle-income countries to expand national transfusion services and blood management systems. Oncology is a major user of blood transfusion but if countries don't have the capacity to manage the bulk of oncology, it will limit complex surgery options.

GIOSTAR/HEAMGEN has acquired the exclusive license to the patent for the technique for stem cell proliferation from University of California San Diego (UCSD). The founding team of GIOSTAR/HEAMGEN is comprised of the scientists and clinicians who were involved in creating the Intellectual Property at UCSD and has already achieved PROOF OF CONCEPT - the optimized lab scale proliferation of mature red blood cells - at UCSD as part of their research.

GIOSTAR/HEAMGEN is currently looking for strategic partnerships (Contact Doug@DMProductionsLLC.com) to accelerate the development of donor-independent red blood cells manufacturing capabilities and advance the proof of concept work already done (patented) around the manufacture of safe, universal donor, human red blood cells. GIOSTAR/HEAMGEN will also develop a full automated proprietary bioreactor using robotic technology to produce abundant quantities of red blood cells with a goal for cost-effective commercialization of fresh, human, universal donor Red Blood Cells (RBCs).

ABOUT GIOSTAR

Dr. Anand Srivastava is a Chairman and Cofounder of California based Global Institute of Stem Cell Therapy and Research (GIOSTAR) headquartered in San Diego, California, (U.S.A.). The company was formed with the vision to provide stem cell based therapy to aid those suffering from degenerative or genetic diseases around the world such as Parkinson's, Alzheimer's, Autism, Diabetes, Heart Disease, Stroke, Spinal Cord Injuries, Paralysis, Blood Related Diseases, Cancer and Burns. GIOSTAR is a leader in developing most advance stem cell based technology, supported by leading scientists with the pioneering publications in the area of stem cell biology. Company's primary focus is to discover and develop a cure for human diseases with the state of the art unique stem cell based therapies and products. The Regenerative Medicine provides promise for treatments of diseases previously regarded as incurable.

GIOSTAR is world's leading Stem cell research company involved with stem cell research work for over a decade. It is headed by Dr Anand Srivastava, who is a pioneer and a world-renowned authority in the field of Stem Cell Biology, Cancer and Gene therapy. Several governments and organizations including USA, India, China, Turkey, Kuwait, Thailand, Philippines, Bahamas, Saudi Arabia and many others seek his advice and guidance on drafting their strategic and national policy formulations and program directions in the area of stem cell research, development and its regulations. Under his creative leadership, a group of esteemed scientists and clinicians have developed and established Stem Cell Therapy for various types of autoimmune diseases and blood disorders, which are being offered to patients in USA and soon it will be offered on a regular clinical basis to the people around the globe.

For the original version on PRWeb visit: https://www.prweb.com/releases/giostar_announces_medical_breakthrough_in_biotechnology_and_lifesciences_to_manufacture_abundant_safe_red_blood_cells_from_stem_cells/prweb16854975.htm

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GIOSTAR Announces Medical Breakthrough in Biotechnology and Lifesciences To Manufacture Abundant, Safe Red Blood Cells From Stem Cells - Benzinga

Spinal Cord Stem Cells Comments Off on GIOSTAR Announces Medical Breakthrough in Biotechnology and Lifesciences To Manufacture Abundant, Safe Red Blood Cells From Stem Cells – Benzinga | January 30th, 2020

As amphibians go, axolotls are pretty cute. These salamanders sport a Mona Lisa half-smile and red, frilly gills that make them look dressed up for a party. You might not want them at your soiree, though: Theyre also cannibals. While rare now in the wild, axolotls used to hatch en masse, and it was a salamander-eat-salamander world. In such a harsh nursery, they evolved or maybe kept the ability to regrow severed limbs.

Their regenerative powers are just incredible, says Joshua Currie, a biologist at the Lunenfeld-Tanenbaum Research Institute in Toronto whos been studying salamander regeneration since 2011. If an axolotl loses a limb, the appendage will grow back, at just the right size and orientation. Within weeks, the seam between old and new disappears completely.

And its not just legs: Axolotls can regenerate ovary and lung tissue, even parts of the brain and spinal cord.

Unlike many amphibians, axolotls do not undergo metamorphosis. They stay in their aquatic, larval form (complete with frilly gills) even as they become sexually mature. They can regrow lost limbs, again and again, making them appealing to scientists who want to understand regeneration.

CREDIT: MARK LEAVER, PHD

The salamanders exceptional comeback from injury has been known for more than a century, and scientists have unraveled some of its secrets. It seals the amputation site with a special type of skin called wound epithelium, then builds a bit of tissue called the blastema, from which sprouts the new body part. But until recently, the fine details of the cells and molecules needed to create a leg from scratch have remained elusive.

With the recent sequencing and assembly of the axolotls giant genome, though, and the development of techniques to modify the creatures genes in the lab, regeneration researchers are now poised to discover those details. In so doing, theyll likely identify salamander tricks that could be useful in human medicine.

Already, studies are illuminating the cells involved, and defining the chemical ingredients needed. Perhaps, several decades from now, people, too, might regrow organs or limbs. In the nearer future, the findings suggest possible treatments for ways to promote wound-healing and treat blindness.

The idea of human regeneration has evolved from an if to a when in recent decades, says David Gardiner, a developmental biologist at the University of California, Irvine. Everybody now is assuming that its just a matter of time, he says. But, of course, theres still much to do.

In a working limb, cells and tissues are like the instruments in an orchestra: Each contributes actions, like musical notes, to create a symphony. Amputation results in cacophony, but salamanders can rap the conductors baton and reset the remaining tissue back to order and all the way back to the symphonys first movement, when they first grew a limb in the embryo.

The basic steps are known: When a limb is removed, be it by hungry sibling or curious experimenter, within minutes the axolotls blood will clot. Within hours, skin cells divide and crawl to cover the wound with a wound epidermis.

Next, cells from nearby tissues migrate to the amputation site, forming a blob of living matter. This blob, the blastema, is where all the magic happens, said Jessica Whited, a regenerative biologist at Harvard University, in a presentation in California last year. It forms a structure much like the developing embryos limb bud, from which limbs grow.

This movie shows immune cells, labeled to glow green, moving within a regenerating axolotl fingertip. Scientists know that immune cells such as macrophages are essential for regeneration: When they are removed, the process is blocked.

CREDIT: JOSH CURRIE

Finally, cells in the blastema turn into all the tissues needed for the new limb and settle down in the right pattern, forming a tiny but perfect limb. This limb then grows to full size. When all is done, you cant even tell where the amputation occurred in the first place, Whited tells Knowable Magazine.

Scientists know many of the molecular instruments, and some of the notes, involved in this regeneration symphony. But its taken a great deal of work.

As Currie started as a new postdoc with Elly Tanaka, a developmental biologist at the Research Institute of Molecular Pathology in Vienna, he recalls wondering, Where do the cells for regeneration come from? Consider cartilage. Does it arise from the same cells as it does in the developing embryo, called chondrocytes, that are left over in the limb stump? Or does it come from some other source?

To learn more, Currie figured out a way to watch individual cells under the microscope right as regeneration took place. First, he used a genetic trick to randomly tag the cells he was studying in a salamander with a rainbow of colors. Then, to keep things simple, he sliced off just a fingertip from his subjects. Next, he searched for cells that stuck out say, an orange cell that ended up surrounded by a sea of other cells colored green, yellow and so on. He tracked those standout cells, along with their color-matched descendants, over the weeks of limb regeneration. His observations, reported in the journal Developmental Cell in 2016, illuminated several secrets to the regeneration process.

Regenerative biologist Joshua Currie labeled the cells in axolotls with a rainbow of colors, so that he could follow their migration after he amputated the tip of the salamanders fingertips. In this image, three days after amputation, the skin (uncolored) has already covered the wound.

CREDIT: JOSH CURRIE

For one thing, cell travel is key. Cells are really extricating themselves from where they are and crawling to the amputation plane to form this blastema, Currie says. The distance cells will journey depends on the size of the injury. To make a new fingertip, the salamanders drew on cells within about 0.2 millimeters of the injury. But in other experiments where the salamanders had to replace a wrist and hand, cells came from as far as half a millimeter away.

More strikingly, Currie discovered that contributions to the blastema were not what hed initially expected, and varied from tissue to tissue. There were a lot of surprises, he says.

Chondrocytes, so important for making cartilage in embryos, didnt migrate to the blastema (earlier in 2016, Gardiner and colleagues reported similar findings). And certain cells entering the blastema pericytes, cells that encircle blood vessels were able to make more of themselves, but nothing else.

The real virtuosos in regeneration were cells in skin called fibroblasts and periskeletal cells, which normally surround bone. They seemed to rewind their development so they could form all kinds of tissues in the new fingertip, morphing into new chondrocytes and other cell types, too.

To Curries surprise, these source cells didnt arrive all at once. Those first on the scene became chondrocytes. Latecomers turned into the soft connective tissues that surround the skeleton.

How do the cells do it? Currie, Tanaka and collaborators looked at connective tissues further, examining the genes turned on and off by individual cells in a regenerating limb. In a 2018 Science paper, the team reported that cells reorganized their gene activation profile to one almost identical, Tanaka says, to those in the limb bud of a developing embryo.

Muscle, meanwhile, has its own variation on the regeneration theme. Mature muscle, in both salamanders and people, contains stem cells called satellite cells. These create new cells as muscles grow or require repair. In a 2017 study in PNAS, Tanaka and colleagues showed (by tracking satellite cells that were made to glow red) that most, if not all, of muscle in new limbs comes from satellite cells.

If Currie and Tanaka are investigating the instruments of the regeneration symphony, Catherine McCusker is decoding the melody they play, in the form of chemicals that push the process along. A regenerative biologist at the University of Massachusetts Boston, she recently published a recipe of sorts for creating an axolotl limb from a wound site. By replacing two of three key requirements with a chemical cocktail, McCusker and her colleagues could force salamanders to grow a new arm from a small wound on the side of a limb, giving them an extra arm.

Using what they know about regeneration, researchers at the University of Massachusetts tricked upper-arm tissue into growing an extra arm (green) atop the natural one (red).

CREDIT: KAYLEE WELLS / MCCUSKER LAB

The first requirement for limb regeneration is the presence of a wound, and formation of wound epithelium. But a second, scientists knew, was a nerve that can grow into the injured area. Either the nerve itself, or cells that it talks to, manufacture chemicals needed to make connective tissue become immature again and form a blastema. In their 2019 study in Developmental Biology, McCusker and colleagues guided by earlier work by a Japanese team used two growth factors, called BMP and FGF, to fulfill that step in salamanders lacking a nerve in the right place.

The third requirement was for fibroblasts from opposite sides of a wound to find and touch each other. In a hand amputation, for example, cells from the left and right sides of the wrist might meet to correctly pattern and orient the new hand. McCusckers chemical replacement for this requirement was retinoic acid, which the body makes from vitamin A. The chemical plays a role in setting up patterning in embryos and has long been known to pattern tissues during regeneration.

In their experiment, McCuskers team removed a small square of skin from the upper arm of 38 salamanders. Two days later, once the skin had healed over, the researchers made a tiny slit in the skin and slipped in a gelatin bead soaked in FGF and BMP. Thanks to that cocktail, in 25 animals the tissue created a blastema no nerve necessary.

About a week later, the group injected the animals with retinoic acid. In concert with other signals coming from the surrounding tissue, it acted as a pattern generator, and seven of the axolotls sprouted new arms out of the wound site.

The recipe is far from perfected: Some salamanders grew one new arm, some grew two, and some grew three, all out of the same wound spot. McCusker suspects that the gelatin bead got in the way of cells that control the limbs pattern. The key actions produced by the initial injury and wound epithelium also remain mysterious.

Its interesting that you can overcome some of these blocks with relatively few growth factors, comments Randal Voss, a biologist at the University of Kentucky in Lexington. We still dont completely know what happens in the very first moments.

If we did know those early steps, humans might be able to create the regeneration symphony. People already possess many of the cellular instruments, capable of playing the notes. We use essentially the same genes, in different ways, says Ken Poss, a regeneration biologist at the Duke University Medical Center in Durham who described new advances in regeneration, thanks to genetic tools, in the 2017 Annual Review of Genetics.

Regeneration may have been an ability we lost, rather than something salamanders gained. Way back in our evolutionary past, the common ancestors of people and salamanders could have been regenerators, since at least one distant relative of modern-day salamanders could do it. Paleontologists have discovered fossils of 300-million-year-old amphibians with limb deformities typically created by imperfect regeneration. Other members of the animal kingdom, such as certain worms, fish and starfish, can also regenerate but its not clear if they use the same symphony score, Whited says.

These fossils suggest that amphibians called Micromelerpeton were regenerating limbs 300 million years ago. Thats because the fossils show deformities, such as fused bones, that usually occur when regrowth doesnt work quite right.

CREDIT: NADIA B. FRBISCHET AL / PROCEEDINGS OF THE ROYAL SOCIETY B 2014

Somewhere in their genomes, all animals have the ability, says James Monaghan, a regeneration biologist at Northeastern University in Boston. After all, he points out, all animals grow body parts as embryos. And in fact, people arent entirely inept at regeneration. We can regrow fingertips, muscle, liver tissue and, to a certain extent, skin.

But for larger structures like limbs, our regeneration music falls apart. Human bodies take days to form skin over an injury, and without the crucial wound epithelium, our hopes for regeneration are dashed before it even starts. Instead, we scab and scar.

Its pretty far off in the future that we would be able to grow an entire limb, says McCusker. I hope Im wrong, but thats my feeling.

She thinks that other medical applications could come much sooner, though such as ways to help burn victims. When surgeons perform skin grafts, they frequently transfer the top layers of skin, or use lab-grown skin tissue. But its often an imperfect replacement for what was lost.

Thats because skin varies across the body; just compare the skin on your palm to that on your calf or armpit. The tissues that help skin to match its body position, giving it features like sweat glands and hair as appropriate, lie deeper than many grafts. The replacement skin, then, might not be just like the old skin. But if scientists could create skin with better positional information, they could make the transferred skin a better fit for its new location.

Monaghan, for his part, is thinking about regenerating retinas for people who have macular degeneration or eye trauma. Axolotls can regrow their retinas (though, surprisingly, their ability to regenerate the lens is limited to hatchlings). He is working with Northeastern University chemical engineer Rebecca Carrier, whos been developing materials for use in transplantations. Her collaborators are testing transplants in pigs and people, but find most of the transplanted cells are dying. Perhaps some additional material could create a pro-regeneration environment, and perhaps axolotls could suggest some ingredients.

Carrier and Monaghan experimented with the transplanted pig cells in lab dishes, and found they were more likely to survive and develop into retinal cells if grown together with axolotl retinas. The special ingredient seems to be a distinct set of chemicals that exist on axolotl, but not pig, retinas. Carrier hopes to use this information to create a chemical cocktail to help transplants succeed. Even partially restoring vision would be beneficial, Monaghan notes.

Thanks to genetic sequencing and modern molecular biology, researchers can continue to unlock the many remaining mysteries of regeneration: How does the wound epithelium create a regeneration-promoting environment? What determines which cells migrate into a blastema, and which stay put? How does the salamander manage to grow a new limb of exactly the right size, no larger, no smaller? These secrets and more remain hidden behind that Mona Lisa smile at least for now.

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Regeneration: The amphibian's opus - Knowable Magazine

Skin Stem Cells Comments Off on Regeneration: The amphibian’s opus – Knowable Magazine | January 30th, 2020

SAN FRANCISCO, Jan. 29, 2020 /PRNewswire/ -- The story of Alzheimer's disease is familiar and heartbreaking. As neurons degenerate and die, patients slowly lose their memories, their thinking skills, and ultimately, their ability to perform basicday-to-day tasks.

For years, clinical trials investigating potential treatments for Alzheimer's disease have come up short. That's why researchers at Gladstone Institutes are delving deeper into the question of what drives this complex disease.

Now, a team led by Senior Investigator and President EmeritusRobert Mahley, MD, PhD, has received $4.8 million from the National Institutes of Health (NIH) to study a promising culprit: apoE4, a protein associated with increased risk of Alzheimer's disease.

ApoE4 is one of the forms of apolipoprotein E, a protein that aids repair processes in neurons injured by aging, stroke, or other causes. The most common form is called apoE3, but apoE4 is not rare: it is found in one-quarter of the human population and in about two-thirds of all Alzheimer's patients, which makes it the most important genetic risk factor for the disorder.

"ApoE4 dramatically rewires cellular pathways in neurons and impairs their function," Mahley said. "Our goal is to understand how this rewiring occurs and identify potential new treatment strategies to negate the detrimental effects."

ApoE3 and apoE4 differ at only a single point in the sequence of their amino acid building blocks. But that single change gives apoE4 a very different shape from apoE3, making it more susceptible to being broken down into smaller fragments within a neuron.

"Our work suggests that these apoE4 fragments are toxic to neurons and cause sweeping changes to the collection of proteins expressed within a neuron," Mahley said. "We suspect that their toxicity may underlie much of the neurodegeneration seen in Alzheimer's disease."

A Powerful Partnership

With the new NIH funding, Mahley hopes to illuminate the specifics of apoE4's toxicity in unprecedented molecular detail. Key to this work is his new partnership with Senior InvestigatorNevan Krogan, PhD, and Gladstone Mass Spectrometry Facility Director Danielle Swaney, PhD, who together have extensive expertise in studying how proteins interact with each other.

To get to the bottom of apoE4's impact, they will use a technique called affinity purification mass spectrometry (AP-MS)to first determine which proteins, out of the thousands found in a single cell, interact directly with apoE4 fragments.

"AP-MS is an important first step because it will allow us to define physical interactions between proteins that may underlie the functional deficits observed in neurons that express apoE4," Swaney said. The AP-MS work will be performed in mouse-derived neuronal cells that are similar to human neurons.

In addition to AP-MS, the collaborators will use other advanced protein analysis techniques perfected in Krogan's lab to better understand the cellular processes that are dysregulated in apoE4-expressing neurons. This additional protein work will be performed in neurons derived from human induced pluripotent stem (hiPS) cells. These stem cells are produced from human skin cells, using the procedure developed byShinya Yamanaka, MD, PhD, a Gladstone senior investigator and 2012 Nobel prize winner.

"We are quite excited to be involved in this project," Krogan said. "My lab has successfully applied AP-MS and other cutting-edge proteomic and genetic techniques to many different diseases, and we now hope to enable a much deeper understanding of apoE4."

When combined, results from the APMS work and the additional protein analyses will reveal a list of key proteins involved in processes that are specifically altered in apoE4 neurons compared to apoE3 neurons.

From that list, Mahley and Swaney will select top candidates for further investigation in neurons grown from hiPS cells. Senior InvestigatorYadong Huang, MD, PhD, who has also studied apoE4 extensively, will provide guidance on the use of the hiPS cells.

Using a gene-editing tool called CRISPR, the researchers will see if they can reverse the detrimental effects of apoE4 by activating or inhibiting genes that control their top candidate proteins in the hiPS cell-derived neurons. Finally, they will validate the findings in mice.

"By the end of the project, we hope to narrow down our list to just a few target genes or proteins that protect or restore neuronal health when we activate or inhibit them in live mice with the apoE4 gene," Swaney said. "They could then be explored as potential targets for Alzheimer's treatment in humans."

New Hope for Alzheimer's Disease

Mahley and Swaney already have some ideas about where this work may lead. Earlier this year,they publishedevidence that apoE4 broadly impacts the mitochondriaorganelles that produce the energy that powers a celland perturbs normal energy production.

"Anything could be a target at this point, but I'm particularly interested in the possibility of small-molecule drugs that could protect mitochondria from toxic apoE4 fragments," Mahley said.

Still, mitochondria are just one aspect of the bigger picture. Mahley suspects that what we call "Alzheimer's disease" is actually a collection of related conditions with different underlying causes for different patients.

"Ultimately, I think the treatment of Alzheimer's disease will be similar to the treatment of high blood pressure, in that two, three, sometimes four drugs are needed to control the disorder," he said. "So, we may need a mitochondrial protector, we may need a drug that will correctapoE4's shapeso that it is more like apoE3, and more."

Understanding the complex effects of apoE4as well as the other Alzheimer's disease-associated factorsbeing explored at Gladstonecould one day enable just such a comprehensive approach.

Media Contact:Megan McDevittmegan.mcdevitt@gladstone.ucsf.edu415.734.2019

Related Images

team-of-researchers-who-received.jpg Team of Researchers who Received the Grant Gladstone Senior Investigator and President Emeritus Bob Mahley (center) will collaborate with the director of the Gladstone Mass Spectrometry Facility, Danielle Swaney (left), and Senior Investigator Nevan Krogan (right) to uncover the mechanisms of apoE4 toxicity in Alzheimer's disease.

Related Links

Gladstone Release

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Gladstone Scientists Funded by NIH to Dive Deep Into ApoE4's Role in Alzheimer's Disease - P&T Community

Skin Stem Cells Comments Off on Gladstone Scientists Funded by NIH to Dive Deep Into ApoE4’s Role in Alzheimer’s Disease – P&T Community | January 30th, 2020

(MENAFN - GetNews) Primary Cells Market: Information by Source (Hematopoietic Cells, Skin Cells, Gastrointestinal Cells, Liver Cells, Lung Cells, and Skeletal and Muscle Cells) Type (Human Primary Cells and Animal Primary Cells), End User (Pharmaceutical and Biotechnology Companies and Research Institutes) and Region - Forecast till 2025

Market Highlights

Primary Cells Market is expected to register a CAGR of8.13% during the forecast period, with a market value of USD 1,233.67 Million till 2025. Primary human cells are isolated directly from normal human tissue or blood cells via the enzymatic or mechanical method. Primary cells retain their fundamental cellular functions. Hence, their use in cell-based research programs is increasing significantly.

Numerous factors such as rapid growth in the biotechnology and biopharmaceutical industries, growing cancer research, rising adoption of primary cells over cell lines, increasing demand for monoclonal antibodies, and rising healthcare expenditure are anticipated to drive the growth of the market during the forecast period. Additionally, the growing research on personalized therapies and stem cells is likely to contribute to market growth. However, the high cost of advanced primary cells and risk of contamination may hamper the growth of the market. The increasing preference of primary cells in research and development to develop new drug acts as opportunities for the growth of the primary cells market.

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Segment Analysis

The Global Primary Cells Market is segmented into Source, Type, and End User. By source, the market has been segmented into hematopoietic cells, skin cells, gastrointestinal cells, liver cells, lung cells, and skeletal and muscle cells.

Based on type, the market has been segmented into human primary cells and animal primary cells. Based on end user, the market has been segmented into pharmaceutical and biotechnology companies and research institutes.

Regional Analysis

The Global Primary Cells Market, based on region, has been divided into the Americas, Europe, Asia-Pacific, and the Middle East & Africa.

The Americas is expected to hold the largest share of the global primary cells market. This is owing to the increasing prevalence of cancer and growing government funding in research. Also, the key players in the market are engaged in new launches and strategic collaborations to hold their market position. For instance, in January 2017, STEMCELL Technologies Inc. entered into a license agreement with Cincinnati Children's Hospital Medical Center, to commercialize the center's technology for producing gastrointestinal organoids from pluripotent stem cells (PSCs). Thus, all these factors are driving the primary cells market.

The European market holds the second-largest position in the global primary cells market. Factors attributing to the growth of the market include the rising prevalence of lifestyle-associated conditions, and the presence of developed economies such as Germany, the UK, and France boosts the market growth.

Asia-Pacific is estimated to be the fastest-growing region owing to the rising prevalence of chronic and acute diseases such as HIV, cancer, and diabetes, and the development of new infrastructure to support the healthcare industry are expected to drive the market growth.

The primary cells market in the Middle East & Africa is expected to grow at a significant rate owing to the implementation of a new business strategy such as a growing distribution channel, product launch in the untapped market by the healthcare companies increases the market growth in this region.

Key Players

MRFR recognizes the following companies as theKey Players in the Global Primary Cells Market Thermo Fisher Scientific Inc. (US), AllCells (US), American Type Culture Collection (ATCC) (Virginia), Axol Bioscience Ltd (UK), Cell Biologics, Inc. (Chicago), Lonza Group, AG (Switzerland), Merck KGaA (Germany), PromoCell (UK), STEMCELL Technologies Inc. (Canada), ZenBio, Inc. (Research Triangle Park, NC), and among others.

Key Findings of the Study

The Global Primary Cells Market was valued at USD 722.61 Million in 2018, is estimated to grow at USD 1,233.67 Million by 2025 at a CAGR of 8.13% during the assessment period

Asia-Pacific accounted for the largest share of the global market due to the increasing per capita health spending, growing geriatric population base, and developing countries enhance the market growth

Based on source, the hematopoietic cells segment accounted for the largest market share in 2018

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Primary Cells Market Is Expected to Reach Register USD 1233.67 Million at a CAGR of 8.13% By 2025 - MENAFN.COM

Skin Stem Cells Comments Off on Primary Cells Market Is Expected to Reach Register USD 1233.67 Million at a CAGR of 8.13% By 2025 – MENAFN.COM | January 30th, 2020

The 2019 novel coronavirus (2019-nCoV) outbreak has sparked a speedy response, with scientists, physicians, and front-line healthcare professionals analyzing data in real-time in order to share findings and call out misinformation. Today, The Lancet published two new peer-reviewed studies: one which found that the new coronavirus is genetically distinct from human SARS and MERS, related viruses which caused their own outbreaks, and a second which reports clinical observations of 99 individuals with 2019-nCoV.

The first cases of the coronavirus outbreak were reported in late December 2019. In this new study, Nanshan Chen and colleagues analyzed available clinical, demographic, and laboratory data for 99 confirmed coronavirus cases at the Wuhan Jinyintan Hospital between Jan 1 to Jan 20, 2020, with clinical outcomes followed until 25th January.

Chen and colleagues reported that the average age of the 99 individuals with 2019-nCoV is around 55.5 years, where 51 have additional chronic conditions, including cardiovascular and cerebrovascular (blood flow to the brain) diseases. Clinical features of the 2019-nCoV include a fever, cough, shortness of breath, headaches, and a sore throat. 17 individuals went on to develop acute respiratory distress syndrome, resulting in death by multiple organ failure in 11 individuals. However, it is important to note here that most of the 2019-nCoV cases were treated with antivirals (75 individuals), antibiotics (70) and oxygen therapy (75), with promising prognoses, where 31 individuals being discharged as of 25th January.

Based on this sample, the study suggests that the 2019 coronavirus is more likely to affect older men already living with chronic conditions but as this study only includes 99 individuals with confirmed cases, it may not present a complete picture of the outbreak. As of right now, there are over 6,000 confirmed coronavirus cases reported, where a total of 126 individuals have recovered, and 133 have died.

In a second Lancet study, Roujian Lu and their fellow colleagues carried out DNA sequencing on samples, obtained from either a throat swab or bronchoalveolar lavage fluids, from eight individuals who had visited the Huanan seafood market in Wuhan, China, and one individual who stayed in a hotel near the market. Upon sequencing the coronaviruss genome, the researchers carried out phylogenetic analysis to narrow down the viruss likely evolutionary origin, and homology modelling to explore the virus receptor-binding properties.

Lu and their fellow colleagues found that the 2019-nCoV genome sequences obtained from the nine patients were very similar (>99.98% similarity). Upon comparing the genome to other coronaviruses (like SARS), the researchers found that the 2019-nCoV is more closely related (~87% similarity) to two bat-derived SARS-like coronaviruses, but does not have as high genetic similarity to known human-infecting coronaviruses, including the SARS-CoV (~79%) orMiddle Eastern Respiratory Syndrome (MERS) CoV (~50%).

The study also found that the 2019-nCoV has a similar receptor-binding structure like that of SARS-CoV, though there are small differences in certain areas. This suggests that like the SARS-CoV, the 2019-nCoV may use the same receptor (called ACE2) to enter cells, though confirmation is still needed.

Finally, phylogenetic analysis found that the 2019-nCoV belongs to the Betacoronavirus family the same category that bat-derived coronaviruses fall into suggesting that bats may indeed be the 2019-nCoV reservoir. However, the researchers note that most bat species are hibernating in late December, and that no bats were being sold at the Huanan seafood market, suggesting that while bats may be the initial host, there may have been a secondary animal species which transmitted the 2019-nCoV between bats and humans.

Its clear that we can expect new findings from the research community in the coming days as scientists attempt to narrow down the source of the 2019-nCoV.

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Rapid analysis shows that the 2019-nCoV coronavirus resembles viruses from bats - Massive Science

Skin Stem Cells Comments Off on Rapid analysis shows that the 2019-nCoV coronavirus resembles viruses from bats – Massive Science | January 30th, 2020

Early Clinical Data Support ex vivo Hematopoietic Stem Cell Gene Therapy as a Potentially Promising Treatment Option for X-CGD

BOSTON and LONDON, Jan. 29, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX), a global gene therapy leader, today announced that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for OTL-102, the companys ex vivo autologous hematopoietic stem cell (HSC) gene therapy being investigated for the treatment of X-linked chronic granulomatous disease (X-CGD). The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S.

We are pleased to have received this orphan drug designation from the FDA, which recognizes the potential of OTL-102 to address a rare population of patients with X-CGD, a life-threatening disease with a critical unmet need, said Anne Dupraz-Poiseau, Ph.D., chief regulatory officer at Orchard. We are encouraged by the clinical data published to date and are eager to advance OTL-102 development as quickly as possible for patients with X-CGD.

Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval.

Early academic clinical trial data for OTL-102 that was recently published in Nature Medicine demonstrates that ex vivo autologous HSC gene therapy may be a promising approach for the treatment of X-CGD. The letter, which wasled by researchers at the University of California, Los Angeles (UCLA)including Donald B. Kohn, M.D., one of the study's lead investigators and professor of microbiology, immunology and molecular genetics at UCLA and Great Ormond Street Hospital (UK), provides an analysis of safety and efficacy outcomes in nine severely affected patients with X-CGD. At 12 months post-treatment, six of seven surviving patients, all of whom were adults or late adolescents, exceeded the minimum threshold hypothesized in published literature to demonstrate potential clinical benefit, defined as 10% functioning, oxidase-positive neutrophils in circulation and have discontinued preventive antibiotics.1

As previously reported, two pediatric patients died within three months of treatment from complications deemed by the investigators and independent data and safety monitoring board to be related to pre-existing comorbidities due to advanced disease progression and unrelated to OTL-102. Investigators are planning to enroll additional pediatric patients in 2020 to assess outcomes in this patient population. In addition, there is work underway to improve the efficiency of the drug product manufacturing process prior to initiating a registrational study.

Patients with X-CGD experience significantly reduced quality and length of life, and currently must take daily medications that do not eliminate the risk of fatal infections, said Adrian Thrasher, Ph.D., M.D., one of the studys lead investigators and professor of pediatric immunology and Wellcome Trust Principal Research Fellow at UCL Great Ormond Street Institute of Child Health in London. These data demonstrate that OTL-102 has the potential to become a transformative new treatment option for patients with X-CGD with the evaluation of longer follow up and more patients.

About X-CGDX-linked chronic granulomatous disease (X-CGD) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the cytochrome B-245 beta chain (CYBB) gene encoding the gp91phox subunit of phagocytic NADPH oxidase. Because of this genetic defect, phagocytes, or white blood cells, of X-CGD patients are unable to kill bacteria and fungi, leading to chronic, severe infections. The main clinical manifestations of X-CGD are pyoderma, a type of skin infection; pneumonia; colitis; lymphadenitis, an infection of the lymph nodes; brain, lung and liver abscesses; and osteomyelitis, an infection of the bone. Patients with X-CGD typically start to develop infections in the first decade of life, and an estimated 40 percent of patients die by the age of 35.2 The incidence of X-CGD is currently estimated at between 1 in 100,000 and 1 in 400,000 male births.

Story continues

About OTL-102OTL-102 is an ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of X-CGD. The studies are supported by multiple institutions including the California Institute of Regenerative Medicine, the Gene Therapy Resource Program from the National Heart, Lung, and Blood Institute, the National Institute of Allergy and Infectious Diseases Intramural Program, the Wellcome Trust and the National Institute for Health Research Biomedical Research Centres at Great Ormond Street Hospital for Children NHS Foundation Trust, University College London Hospitals NHS Foundation Trust and University College London. Preclinical and clinical development of OTL-102 had originally been initiated by Genethon (Evry, France) and funded by an EU framework 7 funded consortium, NET4CGD, before being licensed to Orchard.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates,the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018, as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

References1Kang et al. Blood. 2010;115(4):783-912van den Berget al. PLoS One. 2009;4(4):e5234

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD) -...

Skin Stem Cells Comments Off on Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD) -… | January 30th, 2020

Now lets look at how the professionals do it.

Here are those same four strategies, as used in first paragraphs by various science journalists who write for The Timess Trilobites column, Science News and Science News for Students.

Via Trilobites:

From Swimming With the Mysterious Sardine Disco Balls of the Philippines:

Thousands to millions of sardines emerge from a coral wall in cobalt waters just a few yards from the shores of Cebu Island in the Philippines. They move in a single undulating cloud of silver that twists, turns, shrinks, expands and wraps itself around any object that gets in its way. At times, it becomes a thundercloud, blocking out the sun or clapping violently as it suddenly flips its formation to evade a predator.

From Your Phone Carries Chemical Clues About You, but There Are Limits to Using Them:

Your phone is pretty much a high-tech bucket of germs. Thousands of microscopic bugs crawl around on its surface. Remnants of dirty, old skin cells smudge its cover. Tiny hairs stick inside its buttons. And your hands have smeared hundreds of chemicals across its surface. The foundation on your face, the antidepressants you take, the shampoo in your shower and even the hard-core mosquito repellent you applied down in Panama four months ago: All of these things leave traces on your hands and phone. Thats why scientists say they can use your phone to learn a lot about your lifestyle.

From The Mucus-Shooting Worm-Snail That Turned Up in the Florida Keys:

Its bright orange and yellow and about as long as your finger. It lives underwater in a limestone tube with an opening at the tip about as wide as a pencil eraser. It glues its home to hard surfaces and stays for the rest of its life. Its a species of worm-snail that may never have been seen before, and somehow it turned up in an artificial reef in the Florida Keys.

From Eight Crossings and 192 Atoms Long: the Tightest Knot Ever Tied:

British scientists have tied the tightest knot ever tied and, as unlikely as it may seem, this is important.

From A Dolphins Recipe for Octopus:

Try having no arms and eating a live octopus thats crawling around on your head with its tentacles. Failure could mean its your last supper. But a population of bottlenose dolphins off the coast of Australia has found a way to do it.

From Searching for a Rectangular Sun Above the Arctic Circle:

Low on the horizon, the sun casts an eerie light on the icy sea. For several hours, the glow transforms the colorless terrain into shades of pink as the sun does not rise or set, but edges to the side traveling in a semicircle before slowly sinking one last time.

I am far north in the Arctic Ocean, and polar winter has just begun.

Via Science News for Students:

From NASAs Parker probe spots rogue waves and magnetic islands on the sun:

Rogue waves. Floating magnetic islands. Charged particle showers. These are just some of the things NASAs Parker Solar Probe witnessed during its first two close encounters with the sun.

From Science is helping kids become math masters:

Math is one four-letter word that leaves many teens anxious and sweaty. The idea of an impending math test might send shivers down their spines. Some kids avoid their homework or at least delay starting it because they find math so daunting. Their minds might even go blank at the sight of test questions, no matter how well they have studied. If this is you, theres some comfort knowing that youre not alone.

From Viewing virtual reality of icy landscapes may relieve pain:

Wearing a headset to play a virtual-reality game is fun. As you move your head around, you can see the scene from different angles. Youre immersed in a fake environment that seems so real. But the power of VR may go well beyond entertainment. It just might help people who suffer from long bouts of pain, a new study finds.

Via Trilobites:

From Watch Bees Surf to Safety on Waves They Create:

If their honey-making and pollination prowess werent enough, theres a new reason to appreciate honeybees: Theyre world-class surfers.

Beyond pollinating flowers, worker bees which are all females are given the job of searching for water to cool their hives. But if they fall into ponds, their wings get wet and cant be used to fly. A team of researchers at the California Institute of Technology found that when bees drop into bodies of water, they can use their wings to generate ripples and glide toward land like surfers who create and then ride their own waves.

Gnarly, right?

From Its a Dirty Job, but Someone Has to Do It and Not Get Eaten:

If you want to run a successful business, its important to provide a valuable service, advertise it well and do your best to get out what you put in. You should also try to make sure your customers dont eat you.

This is especially true if youre a cleaner shrimp. These industrious crustaceans set up cleaning stations grooves in rocks in which they can retreat in tropical coral reefs, where they pick parasites and dead skin off the fish, eels and turtles that seek them out for this purpose.

From Trilobite Fossils Show Conga Line Frozen for 480 Million Years:

You probably dont think twice when you queue up at the grocery store or join a conga line at a wedding. But this type of single-file organization is a sophisticated form of collective social behavior. And as suggested by the childrens song The Ants Go Marching One-By-One, humans are not the only animals that appreciate the value of orderly lines.

But how far back in the history of living things on Earth does this behavior go?

From How to Talk to Fireflies:

As Earth rotates in the summer, fireflies whisper sweet nothings to each other in the most beautiful language never heard. For millions of years the insects have called to one another secretly, using flashes of light like a romantic morse code. With some rather simple technology a light and a battery scientists have been decoding their love notes for years. But recently I learned that you dont have to be an entomologist to try to talk to fireflies.

From This Is What It Looks Like When an Asteroid Gets Destroyed:

The asteroid belt, hanging out between Mars and Jupiter, is not like the cluttered debris field in The Empire Strikes Back. It may contain millions of rocky and metal objects, but the distances separating them are vast, and collisions are rare.

From In the Race to Live on Land, Lichens Didnt Beat Plants:

A lichen is what happens when a fungus hugs an algae and doesnt let go. Its a sweet arrangement: The fungus offers shelter, and algae feed the fungus. Theyre still separate species, but tear them apart and the fungi typically cant survive. So theyve long been studied as a single organism.

Via Science News:

From A tiny switch could redirect light between computer chips in mere nanoseconds:

Microscopic switches that route light signals between computer chips like tiny traffic conductors could help make faster, more efficient electronics.

From Piranhas and their plant-eating relatives, pacus, replace rows of teeth all at once:

When it comes to scary teeth, piranhas bite is among the most fearsome. Their razor-sharp teeth strip preys flesh with the ease of a butchers knife.

From How tardigrades protect their DNA to defy death:

Tardigrades may partly owe their ability to survive outer space to having the molecular equivalent of cotton candy.

Via Trilobites:

From When Water Balloons Hit a Bed of Nails and Dont Pop:

Is it possible to bounce a water balloon off a bed of nails? Surprisingly, yes.

From Watch a Flower That Seems to Remember When Pollinators Will Come Calling:

Can you remember what you did yesterday? If not, you might want to take a lesson from Nasa poissoniana, a star-shaped flowering plant from the Peruvian Andes with an unusual skill set.

From Millions of Ibises Were Mummified. But Where Did Ancient Egypt Get Them?:

The ancient Egyptians left us with plenty of head scratching. How did they actually build the pyramids? Where is Queen Nefertiti buried? Whats inside that mysterious void in the Great Pyramid of Giza?

From How Making Chocolate Is Like Mixing Concrete:

What do chocolate and concrete have in common?

Via Science News:

From Vampire bat friendships endure from captivity to the wild:

Are friendships formed with those we truly like? Or do we settle for whoever happens to be around?

Via Trilobites:

From Fish Depression Is Not a Joke:

Can a fish be depressed? This question has been floating around my head ever since I spent a night in a hotel across from an excruciatingly sad-looking Siamese fighting fish. His name was Bruce Lee, according to a sign beneath his little bowl.

There we were trying to enjoy a complimentary bloody mary on the last day of our honeymoon and there was Bruce Lee, totally still, his lower fin grazing the clear faux rocks on the bottom of his home. When he did finally move, just slightly, I got the sense that he would prefer to be dead.

From My Dinosaurs Jet Lag Helps Explain Why a Time Change Is Hard:

Good morning. Or confusing morning, really. Come Daylight Saving Time each year, people often complain about how thrown off they feel by the shift of an hour.

I thought they were just whiny. That is, until my dinosaur got jet lag and refused to glow.

Since thats not an everyday occurrence, let me explain the dinosaur first, and then Ill get to how my dinosaurs problems may be connected to your own struggles to function over the next few days. (Hint: Its not only the loss of sleep that causes problems.)

From First the Worm Gets in the Bugs Head. Then the Bug Drowns Itself.:

A few years back, Ryan Herbison, then a graduate student in parasitology at the University of Otago, painstakingly collected about 1,300 earwigs and more than 2,500 sandhoppers from gardens and a beach in New Zealand.

Then, he dissected and examined the insides of their heads.

From Taking the Pulse of a Sandstone Tower in Utah:

In 2013, a mutual friend brought Kat Vollinger and Nathan Richman together as rock climbing partners. Within a few years, they were married, and their shared love of climbing led them on adventures around the world. Thats how, in March 2018, they found themselves scaling Castleton Tower, a nearly 400-foot sandstone spire near Moab, Utah, with a seismometer in tow.

Via Science News for Students:

From Dont toss that vape:

Kristen Lewis is the assistant principal at Boulder High School in Colorado. A large cardboard box sits in her office. Its where she tosses the spoils of her ongoing battle with the newest student addiction: vaping. This is what I call the Box of Death, she explains. Inside it is everything that weve confiscated.

From A first: Kids advise hospital researchers on their medical studies:

Paul Croarkin paces in a conference room as he presents a slideshow. It showcases his latest research on depression.

A psychiatrist, he works at the Mayo Clinic, a hospital in Rochester, Minn. And hes excited. Its the first time hes described his research to the hospitals newest advisory board. He really wants the boards opinions and feedback so that he can improve his study.

The board members pay close attention and offer great ideas. After all, thats their job. But they look a little different from most hospital board members. All are children and teens. They make up the only medical pediatric advisory board in the United States.

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Hooking the Reader Right From the Start: The Times Trilobites Column - The New York Times

Skin Stem Cells Comments Off on Hooking the Reader Right From the Start: The Times Trilobites Column – The New York Times | January 30th, 2020

In what is a world-first and potentially the dawn of a new medical technology to treat damaged hearts, scientists in Japan have succeeded in transplanting lab-grown heart cells into a human patient for the first time ever. The procedure is part of a cutting-edge clinical trial hoped to open up new avenues in regenerative medicine, with the treatment to be given to a further nine patients over the coming years.

The clinical trial harnesses the incredible potential of induced pluripotent stem cells (IPSCs), a Nobel Prize-winning technology developed at Kyoto University in 2006. These are created by first harvesting cells from donor tissues and returning them to their immature state by exposing them to a virus. From there, they can develop into essentially any cell type in the body.

Professor Yoshiki Sawa is a cardiac surgeon at Osaka University in Japan, who has been developing a technique to turn IPSCs into sheets of 100 million heart muscle cells, which can be grafted onto the heart to promote regeneration of damaged muscles. This was first tested on pigs and was shown to improve organ function, which led Japans health ministry to conditionally approve a research plan involving human subjects.

The first transplantation of these cells is a huge milestone for the researchers, with the operation taking place earlier this month and the patient now recovering in the general ward of the hospital. The sheets are biodegradable, and once implanted on the surface of the heart are designed to release growth factors that encourage new formation of healthy vessels and boost cardiac function.

The team will continue to monitor the first patient over the coming year, and over the next three years aims to carry out the procedure on a total of 10 patients suffering from ischemic cardiomyopathy, a condition caused by a heart attack or coronary disease that has left the muscles severely weakened.

I hope that [the transplant] will become a medical technology that will save as many people as possible, as Ive seen many lives that I couldnt save, Sawa said at a news conference on Tuesday, according to The Japan Times.

Source: The Japan Times

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Lab-grown heart cells implanted into human patient for the first time - New Atlas

Cardiac Stem Cells Comments Off on Lab-grown heart cells implanted into human patient for the first time – New Atlas | January 30th, 2020

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

To know Untapped Opportunities in the MarketCLICK HERE NOW

One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Get Discount on Latest Report @CLICK HERE NOW

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Request TOC of the Reportfor more Industry Insights @CLICK HERE NOW

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

About TMR Research:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Excerpt from:
Stem Cell Therapy Market Predicted to Accelerate the Growth by 2017-2025 - Jewish Life News

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market Predicted to Accelerate the Growth by 2017-2025 – Jewish Life News | January 30th, 2020

FRESNO, California (KSEE/KGPE) After getting a breast cancer diagnosis, your heart health may be the last thing on your mind. But, if the cancer is in your left breast right over your heart treatment is more difficult. The medical director and the manager of radiation oncology at Community Cancer Institute explain a new and improved technique of radiation therapy that keeps your heart safer during treatment.

Radiation therapy is one of the most common treatments for cancer. It works by damaging genetic material within cancer cellscausing them to die. However, normal cells can also be affected by the radiation.

Alec Beach, the Manager of Radiation Oncology for Community Cancer Institute says, So Surface Guided Radiation Therapy is a new modality relatively new and new to us in the Valley, to help align patients even better and also to provide some techniquesimprovements in accuracy, alignment and also in the breathing cycle of the patient to deliver radiation therapy at the optimal time.

Surface Guided Radiation Therapy or SGRT has been particularly successful in treating breast cancer patients with cancer in the left breast.

Dr. William Silveira the Medical Director of the Department of Radiation Oncology at Community Cancer Institute says, The problem with left sided breast cancer is that the heart is very close to the breast tissue. If we can get the heart out of the way, that helps tremendously. When we monitor the surface of the patient, we can have the patient take a very deep breath, pulling the heart down and out of the way, and therefore we can treat the patient while the heart is essentially completely out of the way, out of the beams, out of harms way.

Its all about timing and the careful placement of SGRT that will minimize the dosage of radiation to the normal tissues while delivering the maximum amount to the cancerous cells.

Theres a significant reduction in the dose received by the heart with this technology. Many of our patients are now surviving, and down the road we dont want them to experience cardiac disease. Radiation therapy for left sided breast cancer can contribute to cardiac disease. So, although it has a tremendous impact on survival and local control for breast cancer, we also have this potential complication down the road. Minimizing the dose to the heart, really saves patients a lot of trouble, said Dr. Silveira.

The possible cardiac risks of radiation therapy are significantly reduced with SGRT treatment.

I think theres a lot of fear regarding radiation therapy and a lot of it has to do with heart disease. I, myself, do worry about heart disease from radiation therapy quite a bit. This technology allows us to reduce the risk of heart disease significantly by essentially taking the heart almost out of the picture. The risk to the heart would be minimalmuch less than 5 percent, Dr. Silveira said.

SGRT helps to keep the heart safe, but can also be used throughout the body.

So SGRT can be used in multiple anatomical sites head and neck treatment in particular. Obviously, were treating the head or neck, the brain or the brain stem areatheres a lot of critical structures in that area and the alignment in that area is crucial so the mm accuracy is crucial so thats a particularly good area that well be implementing this in. But, it can also be used throughout the body, the abdomen, the pelvis for GYN cancer, for example, prostate treatment, basically anything where the surface can be used to align the treatment, said Beach.

Community Cancer Institute is the only one in the Valley using this advanced technology and Beach says the program strives to be second to none.

I would hope that the patients would take away that were here to do the very best that we can for themyes, its technology, but its not just technology for technologys sake, its with an outcome in mind and I want patients to know that we might take a little extra time to treat you, but I think thats worth it, Beach said.

And Dr. Silveira says it takes a collective effort, It takes a lot to implement, however and we have a great team. Our physicist, dosimetrist and therapist all are really fantastic in putting this program together. So its technology plus people.

To learn more about how community medical centers can help you or a loved one in prevention and treatment, visit http://www.CommunityMedical.org/Cancer.

For local, national, and breaking news, and to get weather alerts, download our FREE mobile app from theApple App Storeor the Google Play Store.

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MedWatch Today: Surface Guided Radiation Therapy Protects the Heart During Treatment - YourCentralValley.com

Cardiac Stem Cells Comments Off on MedWatch Today: Surface Guided Radiation Therapy Protects the Heart During Treatment – YourCentralValley.com | January 30th, 2020

National Research (NASDAQ:NRC) and US Stem Cell (OTCMKTS:USRM) are both small-cap business services companies, but which is the better investment? We will contrast the two businesses based on the strength of their profitability, risk, earnings, valuation, analyst recommendations, dividends and institutional ownership.

Risk & Volatility

National Research has a beta of 0.78, indicating that its stock price is 22% less volatile than the S&P 500. Comparatively, US Stem Cell has a beta of 4.87, indicating that its stock price is 387% more volatile than the S&P 500.

Insider and Institutional Ownership

39.7% of National Research shares are held by institutional investors. 4.5% of National Research shares are held by company insiders. Comparatively, 16.7% of US Stem Cell shares are held by company insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a stock is poised for long-term growth.

Valuation & Earnings

This table compares National Research and US Stem Cells top-line revenue, earnings per share (EPS) and valuation.

National Research has higher revenue and earnings than US Stem Cell.

Profitability

This table compares National Research and US Stem Cells net margins, return on equity and return on assets.

Analyst Recommendations

This is a summary of current recommendations and price targets for National Research and US Stem Cell, as provided by MarketBeat.com.

Summary

National Research beats US Stem Cell on 7 of the 9 factors compared between the two stocks.

About National Research

National Research Corporation (NRC) is a provider of analytics and insights that facilitate revenue growth, patient, employee and customer retention and patient engagement for healthcare providers, payers and other healthcare organizations. The Companys portfolio of subscription-based solutions provides information and analysis to healthcare organizations and payers across a range of mission-critical, constituent-related elements, including patient experience and satisfaction, community population health risks, workforce engagement, community perceptions, and physician engagement. The Companys clients range from acute care hospitals and post-acute providers, such as home health, long term care and hospice, to numerous payer organizations. The Company derives its revenue from its annually renewable services, which include performance measurement and improvement services, healthcare analytics and governance education services.

About US Stem Cell

U.S. Stem Cell, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of autologous cellular therapies for the treatment of chronic and acute heart damage, and vascular and autoimmune diseases in the United States and internationally. Its lead product candidates include MyoCell, a clinical therapy designed to populate regions of scar tissue within a patient's heart with autologous muscle cells or cells from a patient's body for enhancing cardiac function in chronic heart failure patients; and AdipoCell, a patient-derived cell therapy for the treatment of acute myocardial infarction, chronic heart ischemia, and lower limb ischemia. The company's product development pipeline includes MyoCell SDF-1, an autologous muscle-derived cellular therapy for improving cardiac function in chronic heart failure patients. It is also developing MyoCath, a deflecting tip needle injection catheter that is used to inject cells into cardiac tissue in therapeutic procedures to treat chronic heart ischemia and congestive heart failure. In addition, the company provides physician and patient based regenerative medicine/cell therapy training, cell collection, and cell storage services; and cell collection and treatment kits for humans and animals, as well operates a cell therapy clinic. The company was formerly known as Bioheart, Inc. and changed its name to U.S. Stem Cell, Inc. in October 2015. U.S. Stem Cell, Inc. was founded in 1999 and is headquartered in Sunrise, Florida.

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Contrasting US Stem Cell (OTCMKTS:USRM) and National Research (OTCMKTS:NRC) - Riverton Roll

Cardiac Stem Cells Comments Off on Contrasting US Stem Cell (OTCMKTS:USRM) and National Research (OTCMKTS:NRC) – Riverton Roll | January 30th, 2020

Research using heart cells from squirrels, mice and people identifies an evolutionary mechanism critical for heart muscle function.

Gene defect that affects a protein found in the heart muscle interferes with this mechanism to cause hypertrophic cardiomyopathy, a potentially fatal heart condition.

Imbalance in the ratio of active and inactive protein disrupts heart muscle's ability to contract and relax normally, interferes with heart muscle's energy consumption.

Treatment with a small-molecule drug restores proper contraction, energy consumption in human and rodent heart cells.

If affirmed in subsequent studies, the results can inform therapies that could halt disease progression, help prevent common complications, including arrhythmias and heart failure.

The heart's ability to beat normally over a lifetime is predicated on the synchronized work of proteins embedded in the cells of the heart muscle.

Like a fleet of molecular motors that get turned on and off, these proteins cause the heart cells to contract, then force them to relax, beat after life-sustaining beat.

Now a study led by researchers at Harvard Medical School, Brigham and Women's Hospital and the University of Oxford shows that when too many of the heart's molecular motor units get switched on and too few remain off, the heart muscle begins to contract excessively and fails to relax normally, leading to its gradual overexertion, thickening and failure.

Results of the work, published Jan. 27 inCirculation, reveal that this balancing act is an evolutionary mechanism conserved across species to regulate heart muscle contraction by controlling the activity of a protein called myosin, the main contractile protein of the heart muscle.

The findings--based on experiments with human, mouse and squirrel heart cells--also demonstrate that when this mechanism goes awry it sets off a molecular cascade that leads to cardiac muscle over-exertion and culminates in the development of hypertrophic cardiomyopathy (HCM), the most common genetic disease of the heart and a leading cause of sudden cardiac death in young people and athletes.

"Our findings offer a unifying explanation for the heart muscle pathology seen in hypertrophic cardiomyopathy that leads to heart muscle dysfunction and, eventually, causes the most common clinical manifestations of the condition," said senior author Christine Seidman, professor of genetics in the Blavatnik Institute at Harvard Medical School, a cardiologist at Brigham and Women's Hospital and a Howard Hughes Medical Institute Investigator.

Importantly, the experiments showed that treatment with an experimental small-molecule drug restored the balance of myosin arrangements and normalized the contraction and relaxation of both human and mouse cardiac cells that carried the two most common gene mutations responsible for nearly half of all HCM cases worldwide.

If confirmed in further experiments, the results can inform the design of therapies that halt disease progression and prevent complications.

"Correcting the underlying molecular defect and normalizing the function of heart muscle cells could transform treatment options, which are currently limited to alleviating symptoms and preventing worst-case scenarios such as life-threatening rhythm disturbances and heart failure," said study first author Christopher Toepfer, who performed the work as a postdoctoral researcher in Seidman's lab and is now a joint fellow in the Radcliffe Department of Medicine at the University of Oxford.

Some of the current therapies used for HCM include medications to relieve symptoms, surgery to shave the enlarged heart muscle or the implantation of cardioverter defibrillators that shock the heart back into rhythm if its electrical activity ceases or goes haywire. None of these therapies address the underlying cause of the disease.

Imbalance in the motor fleet

Myosin initiates contraction by cross-linking with other proteins to propel the cell into motion. In the current study, the researchers traced the epicenter of mischief down to an imbalance in the ratio of myosin molecule arrangements inside heart cells. Cells containing HCM mutations had too many molecules ready to spring into action and too few myosin molecules idling standby, resulting in stronger contractions and poor relaxation of the cells.

An earlier study by the same team found that under normal conditions, the ratio between "on" and "off" myosin molecules in mouse heart cells is around 2-to-3. However, the new study shows that this ratio is off balance in heart cells that harbor HCM mutations, with disproportionately more molecules in active versus inactive states.

In an initial set of experiments, the investigators analyzed heart cells obtained from a breed of hibernating squirrel as a model to reflect extremes in physiologic demands during normal activity and hibernation. Cells obtained from squirrels in hibernation--when their heart rate slows down to about six beats per minute--contained 10 percent more "off" myosin molecules than the heart cells of active squirrels, whose heart rate averages 340 beats per minute.

"We believe this is one example of nature's elegant way of conserving cardiac muscle energy in mammals during dormancy and periods of deficient resources," Toepfer said.

Next, researchers looked at cardiac muscle cells from mice harboring the two most common gene defects seen in HCM. As expected, these cells had altered ratios of "on" and "off" myosin reserves. The researchers also analyzed myosin ratios in two types of human heart cells: Stem cell-derived human heart cells engineered in the lab to carry HCM mutations and cells obtained from the excised cardiac muscle tissue of patients with HCM. Both had out-of-balance ratios in their active and inactive myosin molecules.

Further experiments showed that this imbalance perturbed the cells' normal contraction and relaxation cycle. Cells harboring HCM mutations contained too many "on" myosin molecules and contracted more forcefully but relaxed poorly. In the process, the study showed, these cells gobbled up excessive amounts of ATP, the cellular fuel that sustains the work of each cell in our body. And because oxygen is necessary for ATP production, the mutated cells also devoured more oxygen than normal cells, the study showed. To sustain their energy demands, these cells turned to breaking down sugar molecules and fatty acids, which is a sign of altered metabolism, the researchers said.

"Taken together, our findings map out the molecular mechanisms that give rise to the cardinal features of the disease," Seidman said. "They can help explain how chronically overexerted heart cells with high energy consumption in a state of metabolic stress can, over time, lead to a thickened heart muscle that contracts and relaxes abnormally and eventually becomes prone to arrhythmias, dysfunction and failure."

Restoring balance

Treating both mouse and human heart cells with an experimental small-molecule drug restored the myosin ratios to levels comparable to those in heart cells free of HCM mutations. The treatment also normalized contraction and relaxation of the cells and lowered oxygen consumption to normal levels.

The drug, currently in human trials, restored myosin ratios even in tissue obtained from the hearts of patients with HCM. The compound is being developed by a biotech company; two of the company's co-founders are authors on the study. The company provided research support for the study.

In a final step, the researchers looked at patient outcomes obtained from a database containing medical information and clinical histories of people diagnosed with HCM caused by various gene mutations. Comparing their molecular findings from the laboratory against patient outcomes, the scientists observed that the presence of genetic variants that distorted myosin ratios in heart cells also predicted the severity of symptoms and likelihood of poor outcomes, such as arrhythmias and heart failure, among the subset of people that carried these very genetic variants.

What this means, the researchers said, is that clinicians who identify patients harboring gene variants that disrupt normal myosin arrangements in their heart muscle could better predict these patients' risk of adverse clinical course.

"This information can help physicians stratify risk and tailor follow-ups and treatment accordingly," Seidman said.

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Researchers trace the molecular roots of potentially fatal heart condition - Jill Lopez

Cardiac Stem Cells Comments Off on Researchers trace the molecular roots of potentially fatal heart condition – Jill Lopez | January 28th, 2020

$1 Million in Funding from the USAISR Supporting Collaborative Research Project

Pilot Animal Study Successfully Completed; Larger Preclinical Study Underway

NEW YORK, Jan. 28, 2020 (GLOBE NEWSWIRE) -- MicroCures, a biopharmaceutical company developing novel therapeutics that harness the bodys innate regenerative mechanisms to accelerate tissue repair, today announced the advancement of its ongoing collaborative research project with the United States Army Institute of Surgical Research (USAISR) in the area of burn wound healing. The collaboration, which is being carried out under a Cooperative Research and Development Agreement (CRADA) with the USAISR and supported by $1 million in funding, is focused on evaluating the therapeutic potential of MicroCures lead product candidate, siFi2, in accelerating the healing of burn wounds. siFi2, a small interfering RNA (siRNA) therapeutic that can be applied topically, is designed to enhance recovery after trauma. Following the successful completion of the collaborations initial pilot animal study, MicroCures and the USAISR have initiated a second, larger preclinical burn study of siFi2.

MicroCures technology is based on foundational scientific research at Albert Einstein College of Medicine regarding the fundamental role that cell movement plays as a driver of the bodys innate capacity to repair tissue, nerves, and organs. The company has shown that complex and dynamic networks of microtubules within cells crucially control cell migration, and that this cell movement can be reliably modulated to achieve a range of therapeutic benefits. Based on these findings, the company has established a first-of-its-kind proprietary platform to create siRNA-based therapeutics capable of precisely controlling the speed and direction of cell movement by selectively silencing microtubule regulatory proteins (MRPs).

The company has developed a broad pipeline of therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. Unlike regenerative medicine approaches that rely upon engineered materials or systemic growth factor/stem cell therapeutics, MicroCures technology directs and enhances the bodys inherent healing processes through local, temporary modulation of cell motility. The companys lead drug candidate, siFi2, is a topical siRNA-based treatment designed to silence the activity of Fidgetin-Like 2 (FL2), a fundamental MRP, within an area of wounded tissue. In doing so, the therapy temporarily triggers accelerated movement of cells essential for repair into an injury area. Importantly, based on its topical administration, siFi2 can be applied early in the treatment process as a supplement to current standard of care.

Our ongoing collaboration with the USAISR is progressing well and we greatly value the support that this partnership is providing us as we work to advance siFi2 toward the clinic. To date, our work with the USAISR has resulted in the successful completion of a pilot study of siFi2 in a preclinical burn wound model and the recent initiation of a larger preclinical study in this indication, said Derek Proudian, chief executive officer of MicroCures. This project highlights a deliberate strategy by MicroCures to align with trusted military and government organizations, such as the USAISR, other Department of Defense entities, Federal Agencies, and the National Institutes of Health, to collaboratively support the development of our novel therapeutic platform. We look forward to continuing these relationships and ultimately developing innovative treatments that can provide important therapeutic benefits to those in the military, as well as the broader public.

About MicroCures

MicroCures develops biopharmaceuticals that harness innate cellular mechanisms within the body to accelerate and improve recovery after traumatic injury. MicroCures has developed a first-of-its-kind therapeutic platform that precisely controls the rate and direction of cell migration, offering the potential to deliver powerful therapeutic benefits for a variety of large and underserved medical applications.

MicroCures has developed a broad pipeline of novel therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. The companys lead therapeutic candidate, siFi2, targets excisional wound healing, a multi-billion dollar market inadequately served by current treatments. Additional applications for the companys cell migration accelerator technology include dermal burn repair, corneal burn repair, cavernous nerve repair/regeneration, spinal cord repair/regeneration, and cardiac tissue repair. Cell migration decelerator applications include combatting cancer metastases and fibrosis. The company protects its unique platform and proprietary therapeutic programs with a robust intellectual property portfolio including eight issued or allowed patents, as well as eight pending patent applications.

Story continues

For more information please visit: http://www.microcures.com

Contact:

Vida Strategic Partners (On behalf of MicroCures)

Stephanie Diaz (investors)415-675-7401sdiaz@vidasp.com

Tim Brons (media)415-675-7402tbrons@vidasp.com

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MicroCures Advances Burn Wound Healing Program Under Cooperative Research and Development Agreement (CRADA) with the U.S. Army Institute of Surgical...

Cardiac Stem Cells Comments Off on MicroCures Advances Burn Wound Healing Program Under Cooperative Research and Development Agreement (CRADA) with the U.S. Army Institute of Surgical… | January 28th, 2020

On Monday, researchers from Japan's Osaka University announced the successful completion of a first-of-its-kind heart transplant.

Rather than replacing their patient's entire heart with a new organ, these researchers placed degradable sheets containing heart muscle cells onto the heart's damaged areas - and if the procedure has the desired effect, it could eventually eliminate the need for some entire heart transplants.

To grow the heart muscle cells, the team started with induced pluripotent stem (iPS) cells. These are stem cells that researchers create by taking an adult's cells - often from their skin or blood - and reprogramming them back into their embryonic-like pluripotent state.

At that point, researchers can coax the iSP cells into becoming whatever kind of cell they'd like. In the case of this Japanese study, the researchers created heart muscle cells from the iSP cells before placing them on small sheets.

The patient who received the transplant suffers from ischemic cardiomyopathy, a condition in which a person's heart has trouble pumping because its muscles don't receive enough blood.

In severe cases, the condition can require a heart transplant, but the team from Osaka University hopes that the muscle cells on the sheet will secrete a protein that helps regenerate blood vessels, thereby improving the patient's heart function.

The researchers plan to monitor the patient for the next year, and they hope to conduct the same procedure on nine other people suffering from the same condition within the next three years.

If all goes well, the procedure could become a much-needed alternative to heart transplants - not only is sourcing iPS cells far easier than finding a suitable donor heart, but a recipient's immune system is more likely to tolerate the cells than a new organ.

"I hope that (the transplant) will become a medical technology that will save as many people as possible, as I've seen many lives that I couldn't save," researcher Yoshiki Sawa said at a news conference, according to The Japan Times.

This article was originally published by Futurism. Read the original article.

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Lab-Grown Heart Muscles Have Been Transplanted Into a Human For The First Time - ScienceAlert

Skin Stem Cells Comments Off on Lab-Grown Heart Muscles Have Been Transplanted Into a Human For The First Time – ScienceAlert | January 28th, 2020

Image: Nur Yucer, PhD, a project scientist, and Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and Professor of Biomedical Sciences and Medicine at Cedars-Sinai. Photo by Cedars-Sinai.

Parkinson's disease is a neurodegenerative disorder that affects predominately dopamine-producing neurons in the brain. Nearly one million will be living with Parkinson's disease in the US this year, according to the Parkinson's Foundation. This is more than the number of people diagnosed with multiple sclerosis, muscular dystrophy and Lou Gehrig's diseasecombined.

About 60,000 Americans are diagnosed with Parkinson's disease each year, and more than 10 million people worldwide are living with it. Incidence of Parkinsons disease increases with age, but an estimated 10 percent of people with Parkinson's disease are diagnosed before age 50. This is called young-onset Parkinson's.

Researchers at Cedars-Sinai, led by Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and Professor of Biomedical Sciences and Medicine at Cedars-Sinai, reported in a study published in Nature Medicine that they found that patients who develop young-onset Parkinsons disease may have been born with dysfunctional brain cells that go undetected for decades.

The research team generated special stem cells, known as induced pluripotent stem cells (iPSCs), from cells of patients suffering from young-onset Parkinsons disease. These iPSCswhich can produce any cell type of the human body, all genetically identical to the patients own cellswere used to produce dopamine neurons from each patient to analyze their functions.

Two key abnormalities were observed in these neurons:

- Dr. Clive Svendsen

After testing a number of drugs on the abnormal dopamine neurons, the researchers discovered that a drug called PEP005 (ingenol mebutate) reduced the elevated levels of alpha-synuclein in both the dopamine neurons in the dish and in laboratory mice. A gel formulation of PEP005 is marketed by LEO Pharma as Picato and is FDA-approved for the treatment of actinic keratosis, a scaly skin patch that develops from years of exposure to the sun. According to the Mayo Clinic, a small percentage of actinic keratosis lesions can eventually become skin cancer.

Michele Tagliati, PhD, Director of the Movement Disorders Program and Vice Chair and Professor in the Department of Neurology at Cedars-Sinai, said the research team next will study how PEP005 might be delivered to the brain and whether or not the abnormalities found in young-onset Parkinson's patients also exist in other forms of Parkinsons.

- Dr. Michele Tagliati.

Edward Kim is Managing Editor of Equities.com.

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Sources: Equities News, Cedars-Sinai

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Cedars-Sinai Study Indicates That Parkinson's Disease May Start Before Birth - Equities.com

Skin Stem Cells Comments Off on Cedars-Sinai Study Indicates That Parkinson’s Disease May Start Before Birth – Equities.com | January 28th, 2020

People living with Type 1 diabetes are certainly faced with some daily hassles, such as finger-prick blood-glucose tests and insulin injections. An Israeli biomedical firm is now stating that such tasks may soon no longer be necessary, however, thanks to its prototype implant.

Developed by Beta-O2 Technologies, the titanium-bodied device is known as the Bio-artificial Pancreas, or the Air for short.

Measuring about 2.5 by 2.5 inches (64 mm), it incorporates a macrocapsule containing live pancreatic cells (aka islets), along with an oxygen tank. The cells can be obtained from a human donor, from the pancreas of a pig, or they can be grown from the patient's own stem cells in a lab. An external port on the oxygen tank allows the patient to refill it with oxygen on a weekly basis.

Once implanted under the skin, the Air is claimed to continuously monitor blood glucose levels, utilizing the oxygen-fed pancreatic cells to produce and deliver insulin whenever necessary. According to the company, the oxygen supply is the key to the device's success other experimental islet-equipped artificial pancreases, which rely on the limited amount of oxygen within the patient's bloodstream, reportedly have difficulty keeping the cells viable.

Additionally, no immunosuppressive treatments are required in order to keep the new implant from being rejected by the body. That said, the company states that it can easily be removed if necessary.

The device has already been trialled on four patients in Sweden, who experienced no side effects after carrying the implant for 10 months the cells remained viable throughout that period. A second-generation version is now being tested on diabetic rats, which have so far maintained normal glucose levels. A larger human trial should begin later this year.

Source: Beta-O2 Technologies

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Artificial pancreas uses oxygen tank to better-produce insulin - New Atlas

Skin Stem Cells Comments Off on Artificial pancreas uses oxygen tank to better-produce insulin – New Atlas | January 28th, 2020