Ribosomes, which build a protein (black) from an RNA strand (blue), may specialize in making particular sets of proteins.

V. ALTOUNIAN/SCIENCE

By Mitch LeslieJun. 21, 2017 , 11:00 AM

The plant that built your computer isn't churning out cars and toys as well. But many researchers think cells' crucial protein factories, organelles known as ribosomes, are interchangeable, each one able to make any of the body's proteins. Now, a provocative study suggests that some ribosomes, like modern factories, specialize to manufacture only certain products. Such tailored ribosomes could provide a cell with another way to control which proteins it generates. They could also help explain the puzzling symptoms of certain diseases, which might arise when particular ribosomes are defective.

Biologists have long debated whether ribosomes specialize, and some remain unconvinced by the new work. But other researchers say they are sold on the finding, which relied on sophisticated analytical techniques. "This is really an important step in redefining how we think about this central player in molecular biology," says Jonathan Dinman, a molecular biologist at the University of Maryland in College Park.

A mammalian cell may harbor as many as 10 million ribosomes, and it can devote up to 60% of its energy to constructing them from RNA and 80 different types of proteins. Although ribosomes are costly, they are essential for translating the genetic code, carried in messenger RNA (mRNA) molecules, into all the proteins the cell needs. "Life evolved around the ribosome," Dinman says.

The standard view has been that a ribosome doesn't play favorites with mRNAsand therefore can synthesize every protein variety. But for decades, some researchers have reported hints of customized ribosomes. For example, molecular and developmental biologist Maria Barna of Stanford University in Palo Alto, California, and colleagues reported in 2011 that mice with too little of one ribosome protein have short tails, sprout extra ribs, and display other anatomical defects. That pattern of abnormalities suggested that the protein shortage had crippled ribosomes specialized for manufacturing proteins key to embryonic development.

Definitive evidence for such differences has been elusive, however. "It's been a really hard field to make progress in," says structural and systems biologist Jamie Cate of the University of California (UC), Berkeley. For one thing, he says, measuring the concentrations of proteins in naturally occurring ribosomes has been difficult.

In their latest study, published online last week in Molecular Cell, Barna and her team determined the abundances of various ribosome proteins with a method known as selected reaction monitoring, which depends on a type of mass spectrometry, a technique for sorting molecules by their weight. When the researchers analyzed 15 ribosomal proteins in mouse embryonic stem cells, they found that nine of the proteins were equally common in all ribosomes. However, four were absent from 30% to 40% of the organelles, suggesting that those ribosomes were distinctive. Among 76 ribosome proteins the scientists measured with another mass spectrometry-based method, seven varied enough to indicate ribosome specialization.

Barna and colleagues then asked whether they could identify the proteins that the seemingly distinctive ribosomes made. A technique called ribosome profiling enabled them to pinpoint which mRNAs the organelles were readingand thus determine their end products. The specialized ribosomes often concentrated on proteins that worked together to perform particular tasks. One type of ribosome built several proteins that control growth, for example. A second type churned out all the proteins that allow cells to use vitamin B12, an essential molecule for metabolism. That each ribosome focused on proteins crucial for a certain function took the team by surprise, Barna says. "I don't think any of us would have expected this."

Ribosome specialization could explain the symptoms of several rare diseases, known as ribosomopathies, in which the organelles are defective. In Diamond-Blackfan anemia, for instance, the bone marrow that generates new blood cells is faulty, but patients also often have birth defects such as a small head and misshapen or missing thumbs. These seemingly unconnected abnormalities might have a single cause, the researchers suggest, if the cells that spawn these different parts of the body during embryonic development carry the same specialized ribosomes.

Normal cells might be able to dial protein production up or down by adjusting the numbers of these specialized factories, providing "a new layer of control of gene expression," Barna says. Why cells need another mechanism for controlling gene activity isn't clear, says Cate, but it could help keep cells stable if their environment changes.

He and Dinman say the use of "state-of-the-art tools" makes the results from Barna's team compelling. However, molecular biologist Harry Noller of UC Santa Cruz doubts that cells would evolve to reshuffle the array of proteins in the organelles. "The ribosome is very expensive to synthesize for the cell," he says. If cells are going to tailor their ribosomes, "the cheaper way to do it" would entail modifying a universal ribosome structure rather than building custom ones.

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There are millions of protein factories in every cell. Surprise, they're not all the same - Science Magazine

PRP Skin Regeneration Therapy, a type of regenerative medicine with the patients own blood, uses components called platelets in the blood to rejuvenate the skin.

Based on an innate wound-healing ability, the therapy is performed by injecting components collected from the blood and is associated with no risk of allergy or infections. The safe therapy has been studied and applied in a variety of fields. It is indicated for a wide variety of conditions; it is used for the treatment of trauma and burn in the department of plastic surgery and also used as an adjunct to implant therapy in the department of dentistry.

Our clinics have introduced a new technology for extraction of platelet-rich plasma containing autologous white blood cells that has a PRP enrichment rate of about 6- to 10-fold (about three to five times higher than that for conventional one). It also contains white blood cells, which are not contained in conventional PRP. The therapy is found to have much greater efficacy in rejuvenating the skin, eliminating wrinkles, and reducing irregularities from acne marks compared with conventional therapy.

Indications: Expected Effects in Cosmetic Medicine

PRP is particularly effective for crepy skin under the eyes (fine wrinkles) that are difficult to treat by conventional Rejuvenation Therapy. Inducing skin regeneration, the therapy is also effective in treating wrinkles, acne marks, sags and wrinkles on the neck.

Features of the Process Include:

1. Allowing the extraction of White Blood Cells not found in conventional PRP

2. The interaction between PRP and white blood cells results in the release of growth factors that potentiate the natural healing power and tissue reorganization potential at injection sites and subsequent regeneration of the skin.

3. In conventional therapy, it takes about two months for any benefit to be seen, although the time varies among individuals. New-PRP Skin Regeneration Therapy produces noticeable symptomatic improvement in a short period of about two weeks.

Comparison with Conventional Procedures

The PRP enrichment rate is three to five times higher than that for conventional procedures, and the time to benefit is reduced to one-fourth.

Mechanism of New-PRP Skin Regeneration Therapy

PRP Skin Regeneration Therapy uses components called platelets contained in the blood. Platelets play a role in stopping bleeding and repairing damaged blood vessels and cells in the body. Platelets contain substances called growth factors that activate and rejuvenate cells in the body. The growth factors, when released, induce the production of collagen and generation of new capillaries to rejuvenate the skin.

Precautions on How to Choose PRP Therapy

Many clinics now use what they call autologous platelets to eliminate wrinkles and treat acne marks. All those clinics claim skin regeneration therapy with autologous platelets. However, the efficacy of therapy differs substantially.

Important factors for greater efficacy include the concentration and component of platelets for injection. As described previously, skin regeneration with PRP is based on the mechanism that platelets release a variety of growth factors to promote tissue repair, angiogenesis, and collagen production for skin rejuvenation.

The efficacy is enhanced by improving the quality of platelets. Our clinics use a method of injecting platelet-rich plasma containing autologous white blood cells that is prepared by mixing enriched platelets (6 to 10 fold) collected from blood and an appropriate amount of white blood cells, which are rarely contained in common PRP. Our method is found to be highly effective in a range of symptoms.

Skin Rejuvenation with PRP

1. Injection of Platelet Component (PRP):The aged skin has less collagen, low elasticity, reduced amount of hyaluronic acid, and low ability to retain moisture.

2. Release of Growth Factors From Platelet Component (PRP) cell growth is activated, and collagen is produced.

3. Regeneration and Rejuvenation of Skin Tissue:Here Collagen is produced, and skin elasticity is improved. The ability to retain moisture is restored.

For separation of platelet-rich plasma (PRP), a dedicated kit called Fibrinet AGF is used. The use of a specific filter and a centrifuge achieves a high platelet recovery rate of 97% or more and allows preparation of plasma containing six to ten times as many platelets as the common one. This is a three step process:

Step 1: Collection of Blood

Step 2:Separation of Platelet Component A specific filter and a centrifuge are used to prepare platelet-rich plasma (PRP) containing autologous white blood cells.

Step 3. Injection of Platelet Component The platelet-rich plasma (PRP) with autologous white blood cells is injected into the area of concern. It takes about 30 to 40 minutes from blood collection to injection.

Platelets and white blood cells exert a synergistic effect, resulting in the release of a variety of growth factors at the injection sites. This promotes the production of collagen and hyaluronic acid and wound healing, leading to improvement of symptoms such as wrinkles and irregularities from acne marks.

Comparison of Conventional Anti-Aging Therapy

PRP Skin Regeneration Therapy is expected to provide great benefit for crepy skin under the eyes, which are difficult to treat with conventional rejuvenating injections and laser therapy. The therapy uses the patients own blood for rejuvenation and thus poses no risk of infection or allergy. It has the advantage of a longer duration of efficacy compared with injection of hyaluronic acid and collagen that are absorbed into the body. Other features such as no need for skin incision and short downtime (swelling usually resolves in two to three hours) make this therapy a safe treatment.

NOTE: When injection is performed under the eyes, redness may persist for two to three days but resolve over time.

Consult a physician about the best procedure, depending on the sites and conditions of your wrinkles and others symptoms.

Before and After

Another feature of the therapy is that the patient will experience a natural change in the operative site, as well as minimal discomfort as the beneficial effects gradually occur after about two weeks of therapy.

As simple as giving a tube of blood, this nonsurgical treatment utilises patients own platelets and stem cells to promote wound healing. PRP can effectively improve the bodys natural collagen production, resulting in a more youthful appearance.

Neocel PRP kits are the only FDA approved stem cells harvesting kit in the world. The Wembley MediSpa in Cape Town is amongst the few clinics in South Africa to offer this world class treatment with a world class Doctor (90 120 mins).

Excerpt from:
Stem Cells PRP, Acne & Skin Rejuvenation Cape Town

Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch occured on Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

View post:
SpaceX launches CU-built heart, bone health experiments to space station - CU Boulder Today

TAMPA Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease amyotrophic lateral sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USFs Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS.

USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

Read this article:
Mice with ALS improve with stem cell therapy - The Ledger

New skin begins to regenerate as soon as 4 days after this has been sprayed on patients, compared to skin grafting surgery which may take weeks of pain and possible infections.

Patients who suffer from burn wounds and scars that cant heal on their own only have 1 option: skin graft surgery. This can be very painful, and usually leads to several other complications, and it takes forever to actually heal.

However, RenovaCare has developed a new breakthrough piece of tech: CellMist, a gun that sprays stem cells into a patients burn wound, effectively allowing healthy skin to grow out of it.

It works literally like we described it as. Within 90 minutes of a patient being brought to the emergency room, they stem cells are isolated, processed, put in a liquid suspension, and then loaded into the CellMist gun. CellMist then gently sprays the stem cells onto the patients burn wound.

Tests conducted in Europe and the US have shown that new skin begins to regenerate as soon as 4 days after its been sprayed on patients, compared to skin grafting surgery which may take weeks of pain and possible infections.

Source: Next Big Future

So far, CellMist has only been used to treat second degree burns. However, evidence has shown that it can be used for other skin wounds and skin disorders. They dont think itll work for third degree burns though, as this kind of burn wound has damaged the entire epidermis and dermis levels. CellMist isnt advanced enough to heal such a deep burn wound, and victims would unfortunately have to stick to more traditional methods of treatment. First degree burn wounds on the other hand only barely touch the epidermis, meaning it can still heal on its own, thus not needing such an expensive piece of technology.

It is good to note that even though there is evidence that it might be able to heal things other than burns, CellMist wasnt built to regenerate skin lost from other kinds of injuries or diseases. Its also pretty limited, because as we stated earlier, it should be used immediately after the burn incident has occurred, or else it wont work.

Its still a pretty handy invention. The reason why skin grafting is so risky is because it involves cutting the skin open and leaving it open for 3-4 weeks. This means that nasty bacteria and fungi can easily get into the open wound within that time, causing several infections and complications.

Source: Next Big Future

With CellMist, however, simply involves extracting a thin layer of the patients healthy skin and stem cells and turning it into a spray, and then distributing the stem cells into the burn wound evenly, without damaging other healthy skin cells. The healing time only takes a few days, so there is little chance for an infection to occur if treated properly. And since the patients own skin cells are used in the process, the regenerated skin looks much more natural, with only little scarring. The stem cells grow into fully functioning layers of skin, from the dermis, to the epidermis, to even blood vessels.

Hopefully, this cool new invention will make way for other forms of stem cell treatments for the reconstruction of other organs, like ones heart and kidneys.

Article Sources:

Deccan Chronicle

Next Big Future

Read more Robotics Engineers are the Future Doctors?

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This Stem Cell Gun Helps Burn Victims Grow New Skin Faster - GineersNow (press release) (registration) (blog)

"I Will Walk Again" The Laura Dominguez Story If there was ever a woman on a mission, its Laura Dominguez. Doctors once told her shed never walk again. And while shes not ready to run a marathon, shes already proving them wrong, with the best yet to come.

An oil spill on a San Antonio freeway is blamed for the car crash that sent Laura and her brother directly into a retaining wall one summer afternoon in 2001. Laura was just 16 years old at the time and the crash left her completely paralyzed from the neck down. Surgeons say she suffered whats known as a C6 vertebrae fracture that severely damaged her spinal cord.

I refused to accept their prognosis that I never would walk again and began searching for other options, says Laura. After stays in several hospitals for nearly a year, Laura and her mother relocated to San Diego, CA so that she could undergo extensive physical therapy. While in California, they met a family whose daughter was suffering from a similar spinal cord injury. They were also looking for other alternatives to deal with spinal cord injuries.

After extensive research and consultations with medical experts in the field of spinal cord injuries, they decided to explore a groundbreaking new surgical procedure using adult stem cells pioneered by Dr. Carlos Lima of Portugal.

The surgery involved the removal of tissue from the olfactory sinus area at the back of the nose--and transplanting it into the spinal cord at the injury site. Both procedures, the harvesting of the tissue and the transplant, were done at the same time. Laura was the tenth person in the world and the second American to have this procedure done and was featured in a special report by PBS called Miracle Cell. (Link to Miracle Cell (PBS) Episode)

Following the surgery she returned to California where she continued with the physical therapy regimen, then eventually returned home to San Antonio. Upon her return home, an MRI revealed her spinal cord was beginning to heal. Approximately 70% of the lesion now looked like normal spinal cord tissue. More importantly to Laura, she began to regain feeling in parts of her upper body and within six months of the surgery regained feeling down to her abdomen.

Improvements in sensory feelings have continued until the present time. She can feel down to her hips, and has regained feeling and some movement in her legs. Lauras upper body has gained more strength and balance and one of the most evident improvements has been her ability to stand and remain standing, using a walker, and with minimal assistance. When she stands she can contract her quadriceps and hamstring muscles.

Every week it seems Im able to do something new, something different that I hadnt done the week before, says Laura.

Now Lauras story is poised to take a new, potentially groundbreaking turn. In the Fall of 2009, she traveled again to Portugal where adult stem cells were extracted from her nose for culturing. As this story is written, she is preparing to fly back to Portugal where scar tissue at her injury site will be removed and her own adult stem cells injected in the area of her original wound.

The Laura Dominguez story is not complete. The next chapter may or may not yield the results she seeksbut no one can deny the determination and courage of Laura. For her part, she has one goal in mind: I will walk again.

We shall update this site and keep you informed on her progress.

The rest is here:
Stem Cell Research Facts - Adult Stem Cell Success

The Falcon 9 rocket is raised at launch pad 39A early Saturday for a second launch attempt. Credit: Spaceflight Now

A Falcon 9 rocket is again standing upright on launch pad 39A at NASAs Kennedy Space Center in Florida after ground teams lowered the booster Friday to swap out mice heading to the International Space Station for medical experiments.

Liftoff is set for 5:07 p.m. EDT (2107 GMT) to begin a nearly two-day journey to the space station, where the Dragon supply ship fixed to the top of the Falcon 9 rocket will arrive Monday.

The Dragon capsule, the first cargo craft SpaceX has refurbished and reused after a previous flight, is carrying nearly 6,000 pounds of experiments and equipment, including 40 mice inside specially-designed transporters for an investigation into a treatment that could combat bone loss in astronauts on long-duration space missions and osteoporosis in patients on the ground.

Once the mice arrive at the space station, astronauts will treat the rodents with NELL-1, a therapeutic treatment designed to promote bone growth, according to Chia Soo, the chief scientist for the experiment and a professor of plastic, reconstructive and orthopaedic surgery at UCLA.

Men and women past the age of 50, on the average, lose about a half-percent of bone mass per year, Soo said. But in microgravity conditions, the astronaut, on average, loses anywhere from 1 to 2 percent of bone mass per month.

She added that bone loss in astronauts has tremendous implications for humans with respect to long-term space travel or space habitation in microgravity because we end up progressively losing bone mass.

Twenty of the mice will return to Earth alive with the SpaceX Dragon supply ship in early July, the first time the commercial spacecraft has landed with live animals on-board. The 20 mice that come back alive will go to UCLAs laboratories for additional research and treatment.

The other 20 mice will remain on the space station for more observation and comparative studies with the mice on Earth. All of the animals will eventually be euthanized.

If successful, this will have tremendous implications for patients on Earth because if you look at statistics approximately one in every two to three females over the age of 50, or one in every four to five males over the age of 50, will have an osteoporosis-related fracture, Soo said.

We are hoping this study will give us some insights on how NELL-1 can work under these extreme conditions and if it can work for treating microgravity-related bone loss, which is a very accelerated, severe form of bone loss, then perhaps it can (be used) for patients one day on Earth who have bone loss due to trauma or due to aging or disease, Soo said.

After the Falcon 9 launch attempts scrub Thursday, teams lowered the launcher at pad 39A and installed a temporary white room on the Dragon capsules hatch to change out the rodent habitats and several other experiments.

The logistics are complicated, as you might imagine,Louis Stodieck, director of BioServe Space Technologies at the University of Colorado Boulder, wrote in an email to Spaceflight Now. We would normally be okay for two back-to-back launch attempts, but because orbital mechanics would not permit a launch attempt (Friday), the first scrub was automatically done for 48 hours rather than 24.

This forced us to reload with new animals and new Transporters (spaceflight habitats for the ride to space for the mice), Stodieck wrote. We plan for additional groups of mice just for such contingencies.

NASA spokesperson Dan Huot said other experiments that required a changeout for the two-day launch delay included a swarm of fruit flies launching to the space station to examine how prolonged spaceflight affects their heart function.

The hearts of the insects beat at about same rate as the human heart, making it a useful analog, scientists said.

We were back in the lab the night of the scrub setting up new egg collections and adult fly vials, said Karen Ocorr, a co-investigator on the fruit fly experiment from theSanford Burnham Research Institute. These replaced the original set of vials and have now been loaded onto the Dragon for todays attempt.

Researchers are sending between 4,000 and 6,000 fruit fly eggs to the space station, where they will hatch before coming back to Earth aboard the Dragon spacecraft.

We would like to understand the role of microgravity on astronaut heart function in order to try to prevent long-term effects when they are in space for long periods and after they come back, Ocorr said.

But there are real-world implications as well for people who are spending long periods of time in bedrest or immobilized, Ocorr said. We expect that what we find in our studies on the ISS will have implications for maintaining cardiac function in those sorts of situations.

Huot said two crystal growth expeiments and a payload to study how microgravity affects cardiac stem cells also needed to be replaced with the two-day launch delay.

Email the author.

Follow Stephen Clark on Twitter: @StephenClark1.

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[ June 3, 2017 ] SpaceX rocket again set for station delivery after scientists swap mice, fruit flies Mission Reports - Spaceflight Now

Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch has been rescheduled for Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

See the rest here:
SpaceX to launch CU-built heart, bone health experiments to space station - CU Boulder Today

A postdoctoral fellow will examine the protein's effects in human cells.

By Lawrence GoodmanJune 1, 2017

In ALS, also known as Lou Gehrigs disease, the bodys motor neurons degenerate and eventually die. As a result, muscles waste away, leading to an inability tospeak, move and, eventually, breathe. Patients typically die within five years of symptom onset.

One possible target for a drug treatment for ALS is the protein TDP-43. Mutations in the gene encoding TDP-43 cause some cases of inherited ALS and almost all sufferers of sporadic ALS to develop clumps of TDP-43 protein intheir neurons.

In recent years, postdoctoral fellow Mugdha Deshpande has been working withassociate professors of biology Avital Rodal and Suzanne Paradis to uncover how the TDP-43 protein damages neurons in model organisms such as the fruit fly Drosophila melanogaster. Now, they want to take the next step and see whether the same effects occur in human cells.

Deshpande is the Blazeman Postdoctoral Fellow for ALS Research, a position funded by the Rhode Island-based Blazeman Foundation for ALS. Based on her discoveries of how TDP-43 affects neurons in model organisms, she recently received a Brandeis Provost Research award to further her research on TDP-43 in human cells.

Deshpandes research focuses on motor neurons, whose nuclei are located in thespinal cord and whose nerve fibers, or axons, stretch throughout the body. In flies, defective TDP-43 has been shown to cause damage in the area where axonsconnect to muscles.

To test whether the same defects occur in humans, Deshpande will utilize a line of induced pluripotent stem cells isolated from an ALS patients skin cells and developed at the University of Massachusetts Medical School. In collaboration with the Human Neuron Core at Boston Children's Hospital, she will transform the stem cells into neurons.

Deshpande plans to study the defects that arise when human neurons develop whileharboring a genetic mutation in the TDP-43 gene. We need to gain an understanding of whats going on, she says. Without that, we are not going to get a therapy for ALS.

See more here:
Looking at the role of the protein TDP-43 in ALS - Brandeis University

Friday November 20 2009

Human embryonic stem cells

Stem cells could create new skin to help burn victims, BBC News reported. It said that French researchers have duplicated the biological steps that occur during skin formation in embryos. This could potentially provide an unlimited source of temporary skin replacements for burn victims while they wait for grafts from their own skin.

The study in mice behind this report used human embryonic stem cells to make keratinocytes (the most common cell types in the skin). These cultured cells were used to create skin equivalents, which grew successfully when they were grafted onto the backs of mice.

This well-conducted research has potentially developed a successful method of culturing tissue in the laboratory that resembles human skin. Only human trials of the technology will show whether such grafts will be accepted (i.e. not rejected by human patients) as permanent transplants or can provide a temporary skin replacement before grafting.

The research was carried out by Dr Hind Guenou and colleagues from the Institute for Stem Cell Therapy and Exploration of Monogenic disease, and BIOalternatives SAS in France along with colleagues in Madrid. The research was funded by the Institut National de la Sant et de la Recherche Mdicale, University Evry Val dEssonne, Association Franaise contre les Myopathies, Fondation Ren Touraine, and Genopole. The authors declare that they have no conflicts of interest and say that the funders had no role in the studys design, analysis or write-up.

The research was published in thepeer-reviewed medical journal the Lancet.

BBC News has covered this research in a balanced way, pointing out that thiswas animal research and that human studies will follow.

This well-conducted research involved laboratory and animal research which investigated whether epidermal stem cells could be cultured in the laboratory and used in skin grafts.

Burn patients are often treated using autologous skin grafts. These involve a section of healthy skin being removed from another part of the body to harvest the patients own skin cells for culture. A graft for the burn site is produced from this culture. There is a delay of about three weeks between the harvesting of the skin and the graft to allow the cells to grow. During this time, the patient is at risk of dehydration and infection.

Having a ready source of skin cells for temporary grafts while patients are waiting for their autologous grafts would improve the outcome of treatment. With this in mind, the researchers investigated whether keratinocytes (the major cell constituent of the outer layer of the skin, or epidermis) could be derived from human embryonic stem cells.

The researchers began by culturing embryonic stem cells in a specialised medium that encourages cell differentiation (the process whereby cells become specialised). Embryonic stem cells can renew themselves and also have the potential to develop into any type of specialised cell.

Cultures of human embryonic stem cells were then grown on a framework made of fibroblast cells and collagen (a fibrous protein that can form a mesh-like structure) made by fibroblasts. Fibroblasts are the cells that form the underlying structure of tissues and are involved in healing.

The stem cells were manipulated so that they developed into epidermal cells, and monitored throughout their specialisation process to make sure the cells were developing into skin cells. The researchers named the cells keratinocytes derived from human embryonic stem cells (K-hESCs).

After several rounds of subculturing and replication, the cells could be frozen and used in further experiments. Bioengineered skin equivalents were then created by growing the K-hESCs on an artificial matrix. These were then grafted onto the backs of five six-week-old immunodeficient female mice. After 10 to 12 weeks, samples were taken from the implants for analysis.

The researchers confirmed thatthe embryonic stem cells differentiated into keratinocytes, which could be grown in culture medium and which replicated well. These derived skin cells were structurally and functionally similar to normal skin cells in that they could be grown on an artificial matrix using classic techniques.

After 12 weeks of growth on immunodeficient mice, the grafted epidermis had developed into a structure that was consistent with mature human skin.

The researchers concluded that their findings build on previous research and show that K-hESCs can develop into a multi-layer epithelium. This epithelium resembles normal human skin both in cell cultures (in vitro) and following grafting onto live animals (in vivo).

They say that growing human skin from human embryonic stem cells could provide an unlimited resource for temporary skin replacement in patients with large burns who are waiting for autologous skin grafts.

If it can be demonstrated that it works in humans, this technology could improve outcomes for burns patients. The researchers report that the first human trial is currently underway.

At present, skin from deceased donors is used to treat burns patients while they wait for their own skin transplant, but there are often problems with rejection. The researchers highlight several potential benefits of an epidermis reconstructed using K-hESCs, including:

It is important to note that, at present, the researchers are only investigating this technology for providing temporary grafts. They say that whether it can be used for permanent grafts for patients who cant use their own cells needs further investigation. They say that for temporary use, the grafts would only be used for the three-week period while the patients permanent graft is grown.

This is a good study and the findings are exciting in this field, but only human research will tell whether it will have a wider application in the treatment of burns patients.

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Skin grafts from stem cells - NHS

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DENVER -- The need is undeniable. The majority of cancer patients in need of a stem cell or bone marrow transplant are not able to get one, in part because they cant find a match.

Doctors hope more people will register to be a donor, and say all it takes to get started is a simple cheek swab.

Paige McCoy, of Parker, did find a match. After she was diagnosed with acute lymphoblastic leukemia at age 28, she needed a stem cell transplant to survive. I honestly thought I was going to die at 28, she said.

But a total stranger absolutely saved her life.

She got to meet her donor for the first time this month at the Gift of Life Gala in New York City. When I saw him I just broke down, because here is basically my hero walking towards me, Paige said.

It was an emotional night. Her donor was a 22 year old student at the University of Tennessee who had registered with a cheek swab at a campus event.

When he agreed to donate, he had to get some shots, then the stem cells were gathered during a type of blood draw. "The blood goes out to the machine. The machine processes the blood, and returns the red blood cells and the rest of the blood products, except for some of the stem cells, back to the donor, said Dr. Michael Maris, the director of research at the Colorado Blood Cancer Institute in partnership with Sarah Cannon Cancer Institute at Presbyterian/St. Lukes.

He says this act, that required no surgery, saved Paiges life.

But Paige knows others werent as lucky. I saw patients that didnt have a donor, and I had a donor and they didnt, and somebody could save their life. Just swab your cheek please. You could really help somebody out, and its so easy, she said.

If you would like to register, you can go to http://www.bethematch.org for cheek swab instructions, or a list of local donation events. Your registration could also help patients needing bone marrow transplants. But the marrow harvesting does require surgery.

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How a simple cheek swab can save a life - FOX31 Denver

Researchers in the U.K. are set to test whether the Zika virus can fight difficult-to-treat brain cancer by attacking its cells, potentially opening up new pathways to treat the aggressive disease. Researchers will focus on glioblastoma, which is the most common form of brain cancer and has a five-year survival rate of 5 percent, Reuters reported.

QUINOA 'MILK' DIET KILLED BABY, AUTHORITIES SAY

The Zika virus causes severe birth defects in an unborn fetus when contracted during pregnancy by attacking developing stem cells in the brain. However, the disease does not have the same devastating effect on fully developed brains, suggesting that if scientists can harness the virus ability to attack the cancer cells, which are similar to developing brain stem cells, healthy brain tissue will go unharmed.

Were taking a different approach, and want to use these new insights to see if the virus can be unleashed against one of the hardest-to-treat cancers, Harry Bulstrode, a lead researcher at Cambridge University, said, in a statement to Reuters.

ITALY VOTES TO MAKE VACCINES MANDATORY

Researchers will use tumor cells in mice to test the virus, and hope that it will slow tumor growth.

If we can learn lessons from Zikas ability to cross the blood-brain barrier and target brain stem cells selectively, we could be holding the key to future treatments, Bulstrode told Reuters.

Active outbreaks of the mosquito-borne illness were reported in at least 51 countries and territories, with pregnant women advised to avoid travel to so-called virus hotbeds. In addition to birth defects, Zika has been associated with neurological disorders including brain and spinal cord infections. Long-term health consequences remain unclear.

Reuters contributed to this report.

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Researchers consider Zika virus for brain cancer treatment - Fox News

Two different groups of researchers have developed ways to generate red and white blood cells in the lab Photograph: Steve Gschmeissner/Getty Images/Science Photo Library RM

How might blood cells be made?

Different groups of researchers say they have developed a way of producing blood cells from human or mouse cells that have been reprogrammed in the lab an advance that has been touted as offering a solution to the need for blood donation. The latest studies are the result of 20 years work in the field.

How exactly did they do it?

The two pieces of work, published at the same time in Nature but by different research groups, take differing approaches. One study, led by George Daley at Harvard Medical School, began with human cells known as induced pluripotent stem cells cells that can make any type of human cell, and which can be produced by genetically reprogramming adult cells, such as those in skin. These were chemically tinkered with to create a tissue that can give rise to blood stem cells and implanted into mice, where blood stem cells were made, which then churned out the different types of cells found in blood, including white blood cells and red blood cells. The approach also worked starting with human embryonic stem cells.

The big achievement is being able to do that transition from a pluripotent stem cell to a blood stem cell, which has never been [done] before, said Cedric Ghevaert of the Cambridge Blood Centre at the University of Cambridge. That is a big story and there is no denying the impact of that.

The other research, led by Raphael Lis, at Weill Cornell Medical College in New York, took a different tack, converting cells taken from the lungs of mice directly into blood stem cells. Once implanted into mice, they too churned out the panoply of blood cells.

Will these breakthroughs remove the need for blood donation?

No. Both of the approaches ultimately produced a collection of different types of blood cells. This, said Ghevaert, is not so useful for transfusions, where particular components of blood, for example, red blood cells, are needed separately. Instead, the research is more relevant for patients who need bone marrow transplants, for example, those withleukaemia.

This is what you get when you get a bone marrow transplant youre given another persons stem cells, said Ghevaert. That has got drawbacks because that other person is never quite a complete match to you, which is why bone marrow transplant is quite a serious procedure. For years we have been asking the question: Could we make the blood stem cells from something else that belongs to the patients that needs those stem cells? he added. This [research] shows the first glimpse of hope. However, there is still some way to go. They have generated enough to transplant a mouse, but if you wanted to transplant a human or indeed produce vast vats of blood cells, you would need an awful lot more and by that, I dont mean even 10 times more, you would need 1,000, 100,000 cells, said Ghevaert. One of biggest problems in this is the manufacturing process, because there is no point in making a pint of blood that costs 1m. As for the second approach, carried out in mice alone, Ghevaert is more cautious. I have seen a lot of very good things done in mice that then dont translate to anything in humans.

Is anyone trying to make blood for transfusions?

Yes, a number of researchers around the world are attempting to manufacture specific components of blood, including Ghevaert, who has been working on using human pluripotent stem cells to produce platelets (the component of blood that helps it to clot).

Is laboratory blood better than donatedblood?

It depends. Blood given in a transfusion has to be of the right type, matching the recipients blood group. For most people, transfusion from donor blood will continue to be the norm such blood is cheap, readily available and safe. But blood manufactured in alaboratory could help some. The only advantage of producing cells in the lab is, for example, to make blood cells that are compatible with patients who are very difficult to transfuse because we simply cant find them a blood group match, said Ghevaert. Donated blood works extremely well for 99.99% of people, therefore I think we have to see these products as a niche product.

Manufactured blood, said Ghevaert, could be a boon in developing countries. If you consider countries where the rate of HIV is 30%, and hepatitis B 60%, finding safe blood is extremely difficult, he said. Manufactured blood in a country where you have endemic viral infections that make the blood supply extremely unsafe, that would be extremely relevant.

Does all this mean that we dont need togive blood any more?

No. A spokesperson for NHS Blood and Transplant said: It will be some time before this research leads to manufactured blood cells being used for patient treatment. Volunteer donors remain a vital lifeblood for patients and will remain so for many years to come.

Link:
Can you manufacture blood cells? - The Guardian

fox6now.com

From hopeless to a miracle: How he got his life back after a crash left him paralyzed fox6now.com "We came to know he would be a good candidate for this regenerative treatment that we offer, meaning the stem cell injection into the spinal cord. ... "He was only the second to receive the stem cells -- at least that dose he received," added Dr. Kurpad.

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From hopeless to a miracle: How he got his life back after a crash left him paralyzed - fox6now.com

An umbilical cord after birth yields about three to five ounces of cell-rich cord blood. That's not a lot, but enough of it can help treat more than 80 or so diseases. A North Texas oncologist says education's key to boosting limited supply.

The KERA Interview with Dr. Sharif

Dr. Suhail Sharif is a surgical oncologist with Texas Health Fort Worth.

Interview Highlights:

Whats special about cord blood: Cord blood has immature blood cells, and you can use these stem cells to, basically, harvest into these patients that have problems with their own blood; for example, because of leukemia or lymphoma or other types of diseases that affect their own blood lines. These can grow into the red blood cells if [they're] deficient or the white blood cells if [they're] deficient or even platelets, for that matter.

Cord blood cells vs. bone marrow cells: Cord blood stem cells are actually stored in a blood bank that you can use on patients that need it. But bone marrow, you actually have to go through a process of harvesting the bone marrow. Its a very painful procedure for whoever is donating the bone marrow. And then they have to go through an extensive and rigorous testing, not only for infectious causes, but also to see if they match with the patient. And then they have to harvest, and they basically have to transplant it. Now, that whole process can take a few months. If you just have cord blood stem cells, these have already been stored and are readily available. And if you have a match with the donor and the recipient, you can use them right away.

Cord blood is limited in supply: If you think about the blood that is in the placenta and the cord, its in the range of three to five ounces. Thats about like half a cup. Thats the reason why you have to gather it from a lot of patients. At this point, there are, I believe, close to 175,000 matched cord blood available."

But its not enough: If you think about what percentage of deliveries actually translate into donating cord blood, its very miniscule. Thats why were educating the parents about the benefits of cord blood so they can donate to a public blood bank so that we can use it in treating patients with deadly cancers and so forth in our community.

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Cord Blood: A Small Amount Does A Lot Of Good - KERA News

12 May 2017 Bone marrow: be someones match A bone-marrow donation is not a scary procedure and doesnt involve drilling into your bones. We found out more.

Unfortunately many people still believe that donating bone marrow means doctors will need to drill into your bones. The reality is the donation process requires no surgery, no general anaesthetic and no drilling.

South African patients who have leukaemia or other blood disorders need a life-saving stem cell transplant and rely on the South African Bone Marrow Registry (SABMR) to find a match.

The challenges faced in growing the SABMR lies in the sentence itself, says Alana James, CEO of the Sunflower Fund. Its called The South African Bone Marrow registry, so when people see the word bone marrow they go Oh no I cant do that, it will hurt!

Understanding bone marrow

Bone marrow is found inside your bones its a soft, fatty tissue that helps with the production of red blood cells (to carry oxygen), white blood cells (to find infection) and platelets (to prevent bleeding). Some of the cells in the bone marrow can be pushed into the blood stream, where it can be collected and then used to help someone in need of a donation.

Real-life hero

Carey Symons, a blood stem cell donor and long-time support of The Sunflower Fund, was on the registry for 10 years before she was called to help a leukaemia patient.

The stats of a perfect match are 1 in 100 000 so you can only imagine my joy in being that 1 in 100 000 and that I was able to contribute to giving someone a second chance.

She travelled from Durban to Mediclinic Constantiaberg in Cape Town where she had a series of painless Neupogen injections that helped stimulate the production and release of blood stem cells.

Three days of injections later she was able to begin the donation process.

1. Two needles (similar to those used when you donate blood) were inserted in each arm. 2. Blood was drawn from one arm and circulated through a cell-separator machine. 3. Her stem cells were collected and the remaining blood was returned through the other arm.

Typically, the donation process takes between four and six hours.

I realised that the day I signed as a donor, I was only hoping to make a difference. I will never know whose life I made a difference to, and part of that mystery excites me. Its a blessing to give without knowing and without being thanked.

Harsh reality

There are just under 74 000 donors on the registry, but at least 400 000 are needed.

We definitely still have a mountain to climb and are committedly doing so. Registering as a donor on the SABMR is a simple process and can be very rewarding, says James. You could be someones perfect match.

There is a 1 in 100 000 chance of being a match. (Image: iStock)

Be someones 1 in 100 000

Signing up to be a donor is simple- if you do meet the criteria, you will receive a reference number and form to fill out. Next, youll go to your nearest Donor Recruitment Clinic where theyll take two test tubes of blood.

Your blood is analysed and put onto the national database. Unfortunately the tissue typing test is expensive (it costs R2 000) but you can make a donation to help free up their funds.

Youll receive your donor card in six to eight weeks, and if youre ever a match for a patient, you will be called.

Read more:

CT teen desperately needs bone marrow transplant

URGENT need for more bone marrow donors in SA

Needed: Black bone marrow donors

A leukaemia patient who had recently undergone a bone marrow transplant received a massive surprise when Taylor Swift visited him n hospital. When she saw that he had a keyboard in his room she asked him to play for her. As he plays Adele's "Someone like you", Taylor joins in, singing along!

CANSAs purpose is to lead the fight against cancer in South Africa. Its mission is to be the preferred non-profit organisation that enables research, educates the public and provides support to all people affected by cancer. Questions are answered by CANSAs Head of Health Professor Michael Herbst and Head of Advocacy Magdalene Seguin. For more information, visit cansa.org.za.

The information provided does not constitute a diagnosis of your condition. You should consult a medical practitioner or other appropriate health care professional for a physical exmanication, diagnosis and formal advice. Health24 and the expert accept no responsibility or liability for any damage or personal harm you may suffer resulting from making use of this content.

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Science Daily

New intervertebral discs from stem cells Science Daily The study on the sick German shepherds was organized as follows: With the permission of the dog owners, neurologist Frank Steffen and his team removed stem cells from the marrow of the pelvic bone of the affected animals. After the cleaning and ...

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New intervertebral discs from stem cells - Science Daily

Science Daily

Engineering human stem cells to model the kidney's filtration barrier on a chip Science Daily ... of kidney diseases and drug toxicities, and the stem cell-derived kidney podocytes we developed could even offer a new injectable cell therapy approach for regenerative medicine in patients with life-threatening glomerulopathies in the future ...

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Engineering human stem cells to model the kidney's filtration barrier on a chip - Science Daily

"These new follow-up results based on MRI scans are very encouraging, and strongly suggest that AST-OPC1 cells have engrafted in these patients post-implantation and have the potential to prevent lesion cavity formation, possibly reducing long-term spinal cord tissue deterioration after spinal cord injury," said Dr. Edward Wirth, Chief Medical Officer of Asterias. "Moreover, these new results add to the overall body of data supporting AST-OPC1's safety, and are consistent with safety data from our previous Phase 1 study in thoracic spinal cord injury and our extensive preclinical studies in more than 3,000 animals."

Under the study protocol, patients are monitored by MRI scans at regular intervals over 12 months in order to assess status of the injection site and surrounding tissues.

The Company will discuss the MRI data in more detail on its first quarter 2017 conference call and webcast on May 11, 2017 at 4:30 p.m. Eastern / 1:30 p.m Pacific. For both "listen-only" participants and those participants who wish to take part in the question-and-answer session, the call can be accessed by dialing 800-533-7619 (U.S./Canada) or 785-830-1923 (international) five minutes prior to the start of the call and providing the Conference ID 7610291. To access the live webcast, go to http://asteriasbiotherapeutics.com/inv_events_presentations.php.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/new-mri-data-from-asterias-ongoing-scistar-clinical-study-indicates-ast-opc1-cells-prevent-formation-of-damaging-lesion-cavities-in-patients-suffering-severe-spinal-cord-injury-300455768.html

SOURCE Asterias Biotherapeutics, Inc.

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New MRI Data from Asterias' Ongoing SCiStar Clinical Study Indicates AST-OPC1 Cells Prevent Formation of ... - PR Newswire (press release)

Motor neurons are the nerve cells in the body responsible for controlling movement. A number of diseases are caused by damage to motor neurons, including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). In order to treat these diseases, scientists are developing methods to generate new, healthy motor neurons from stem cells. A recent study has elucidated the cellular mechanisms that control the motor neuron differentiation, paving the way for new treatments for motor neuron diseases.

Each time we voluntarily move an arm or leg, or when our lungs involuntarily expand and contract, signals from the brain are sent along a chain to the spinal cord, where motor neuron cell bodies reside. These motor neurons terminate in muscle cells, where they transmit the nerve impulses in order to produce muscle contractions. In ALS, there is a progressive destruction of motor neurons due to either a genetic defect or an unknown environmental trigger. Motor neuron damage in ALS leads to progressive muscle weakness that affects all parts of the body, impairing the ability to speak, swallow, and eventually breathe. SMA is caused by gene mutations and is characterized by similarly progressive damage to motor neurons that causes muscle weakness. If respiratory muscles are affected, SMA can be fatal.

Scientists aim to develop gene therapies for these diseases that can repair the damaged motor neurons and improve the functioning and lifespan of patients. To do this, they must first understand the signals that induce motor neuron development from stem cells. Stem cells are the precursors for every type of cell in the body. They are triggered to differentiate into various cell types via cellular signaling molecules called transcription factors, which act on DNA to turn on specific genes. Which genes are turned on will determine the phenotypic fate of each cell. Typically, each cell goes through several stages of development before reaching its final fate.

A group of researchers from several universities recently teamed up to elucidate these programming pathways. They had previously discovered that a group of transcription factors called the NIL factors Ngn2, Isl1, and Lhx3 can induce motor neuron development from embryonic stem cells without passing through any of the intermediate stages. Moreover, the NIL factors achieved the transition to the motor neuron fate with a 90% success rate, and the process took only two days. This so-called direct programming pathway was an exciting finding with respect to clinical applications, because it can be achieved both in vitro and in living organisms at the site of cell damage.

In the current study published in the journal Cell Stem Cell, Esteban Mazzoni and colleagues further investigated the process by which transcription factors bind to and activate parts of DNA during the first 48 hours after NIL expression. First, the researchers used single-cell RNA sequencing (RNA-seq) to study the timing of gene expression after induction by NIL programming factors. RNA-seq is a technique that reveals the presence and quantity of RNA in a sample at a specific point in time. Thus, as transcription factors turn genes on, these genes are transcribed into RNA that can be measured and quantified.

The researchers also studied chromatin remodeling during motor neuron programming. Chromatin is a tightly-packed form of DNA which regulates the expression of genes through changes in its structure. Promoters are regions of the DNA where transcription factors bind in order to initiate gene transcription. Chromatin must undergo structural changes, called remodeling, in order for the DNA to be accessible to transcription factors. Typically, as cells move through the differentiation process, chromatin changes that occur at promoter regions will restrict the differentiation potential of the cell.

To study this chromatin remodeling process, a ChIP-seq time series was performed. ChIP-seq combines chromatin immunoprecipitation with DNA sequencing to identify the binding sites of proteins that associate with DNA. Antibodies against the bound proteins are used to extract protein-DNA complexes, and the DNA binding sites can be sequenced. In addition, the researchers used an assay for transposase-accessible chromatin with high throughput sequencing (ATAC-seq) to study chromatin accessibility. Proteins called transposons incorporate into exposed, or accessible, portions of chromatin. Therefore, identifying the locations of transposons in the DNA can indicate what parts of the DNA are being actively transcribed, or turned on.

This series of experiments revealed information about how genes are turned on and off over the 48-hour process of motor neuron formation. Initially, the transcription factors Ngn2 and Isl1/Lhx3 induce different sets of genes in parallel. Whereas Ngn2 controls genes associated with generic neuronal differentiation, Isl1 and Lhx3 activate genes specific for spinal cord and motor neurons. As programming progresses, Ngn2 induces the expression of two other transcription factors, Ebf and Onecut. These transcription factors modify the chromatin state to enable Isl1/Lhx3 binding to previously inaccessible sites on the DNA that contain the terminal motor neuron genes necessary to complete the programming process.

These experiments showed that the activities of Ngn2 and Isl1/Lhx3 act in tandem to induce direct motor neuron programming from stem cells. The researchers hope to apply these findings clinically. By triggering this programming pathway in the body, cells in the spinal cord can be induced to differentiate into motor neurons, replacing the neurons that are damaged in diseases such as ALS.

References

Czarzasta J., Habich A., Siwek T., Czaplinski A., Maksymowicz W., Wojtikiewicz J. (2017) Stem cells for ALS: an overview of possible therapeutic approaches. Int J Dev Neurosci. DOI: 10.1016/j.ijdevneu.2017.01.003

Farrar M., Park S., Vucic S., Carey K., Turner B., Gillingwater T., Swoboda K., Kiernan M. (2016) Emerging therapies and challenges in Spinal Muscular Atrophy. Ann Neurol. DOI: 10.1002/ana.24864

Mazzoni, E.O., Mahony, S., Closser, M., Morrison, C.A., Nedelec, S., Williams, D.J., An, D., Gifford, D.K., and Wichterle, H. (2013). Synergistic binding of tran- scription factors to cell-specific enhancers programs motor neuron identity. Nat. Neurosci. 16:12191227. DOI:10.1038/nn.3467

Velasco S., Ibrahim M., Kakumanu A., Garipler G., Aydin B., Al-Sayegh M., Hirsekorn A., Abdul-Rahman F., Satija R., Ohler U., Mahony S., Mazzoni, E. (2016) A Multi-step Transcriptional and Chromatin State Cascade Underlies Motor Neuron Programming from Embryonic Stem Cells. Cell Stem Cell. DOI: 10.1016/j.stem.2016.11.006

Image via ColiN00B / Pixabay.

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Motor Neurons Why Are They Important and How Are They Made? - Brain Blogger (blog)