HERE are four beaming children none of whom would be alive today if a stranger had not given blood.

Each of their lives was saved by a transfusion, yet many of us never find the time to sign up to become a donor.

NHS Blood and Transplant is encouraging readers to make giving blood one of their New Year resolutions.

It is particularly calling on men to donate because their blood can be more suitable for treating patients. The families of these four survivors tell Lynsey Hope their stories.

'We worry every day he might suffer a serious bleed'

GEORGE CLAXTON lives with mum Faye, 36, a salon owner, dad Luke, 34, an electrical engineer, and sister Ella, six, in Huntingdon, Cambridgeshire. Faye says:

"When George was 14 months old he was diagnosed with a rare platelet disorder.

"The condition doesnt have a name but it means his blood cant clot properly.

"Tiny blood cells called platelets in his blood are the wrong shape and size and he has to take medication daily.

"We found out he had it after he suffered a virus and came out with a rash.

"Its called petechiae but can look similar to meningitis.

"We took George to A&E at Hinchingbrooke Hospital near Huntington. Blood tests came back negative and we were sent home.

"But two weeks later, we were back again.

"We were referred to specialists at Addenbrookes Hospital in Cambridge, who discovered George was bleeding under the skin.

"Its been hard to accept its a lifelong condition and not something that can be cured.

"There have been two occasions when George has needed a transfusion.

"The first was in June 2016.

"Doctors had to perform a transfusion before he had a tooth extracted to make sure he didnt bleed too much during the procedure.

"In May last year, he fell over in the school playground and hurt his elbow, causing a bleed in his joint.

"George has been brave from the start.

"He loves football but we worry every day he may have an accident that causes a serious bleed.

"He can also have spontaneous bleeds.

"His little sister was also diagnosed with the condition.

"She hasnt needed a transfusion yet but she may do and that is devastating for us as parents.

"Were so grateful to people who donate blood.

"It can enable people to live."

'Just an hour of your time could be the gift of a lifetime'

JESSICA FAY lives in Burnley with her mum Laura Bell, 32, dad Adam Fay, 39, who is a carer, and her brothers Kyle, 14, Denver, 13, Jayden, eight and Taylor, six. Laura, a full-time mum, says:

"Jessica was diagnosed with meningitis and septicaemia when she was 15 weeks old.

"I took her to the GP when she started feeling unwell.

"She wasnt feeding and had a high temperature.

"The doctor was concerned and said I must take her straight to hospital.

"Within hours of arriving at A&E, Jessica stopped breathing and was put on life-support.

"The disease had taken over her body and, one by one, her organs were shutting down.

"There was only one option. A blood transfusion might dilute the infection in her blood and give her a chance.

"There was a risk her body would reject the blood and we knew if that happened wed lose her.

"Incredibly though, that blood transfusion saved her life.

"She remained in intensive care for a week and, after three weeks, she came home.

"Jessica was being given so many treatments in those terrible few weeks that I didnt think too much about where the blood had come from.

"But when she recovered, I realised that without it she would not have made it. Unfortunately, Jessica suffered some brain damage because of what happened.

"She has social communication disorder and finds it hard to make friends.

"She is an incredible child and Im so grateful to whoever it was that took the time to donate blood for her.

"If someone hadnt donated that blood, Jessica would be dead.

"She has done all she can to give something back.

"Shes raised thousands of pounds for charity by organising events in the community.

"I would urge anyone who can to give blood it is just an hour of your time but it could be the gift of a lifetime to a child like Jessica.'

'Our baby can be in a lot of pain due to the disease'

EZRAH PINK was born with sickle cell disease. He lives with his mum Serena, 30, who looks after an office building, and her partner Courtney, 32, an estate agent, in Beckenham, Kent. Serena says:

"We knew before Ezrah was born that he might have sickle cell disease.

"When I was pregnant, doctors found out I carried a gene.

"About a week after he was born, they confirmed Ezrah had the disease.

"People with sickle cell produce unusual C-shaped red blood cells, meaning they sometimes get stuck or block blood vessels. At first, he didnt show any symptoms.

"He started having problems when he was around 11 months.

"Since then its been a whirlwind. We have been in and out of hospital.

"Id never known anyone with sickle cell so its been a tough learning curve and the condition will affect him for life.

"Ezrah has already had four blood transfusions.

"When one of his odd-shaped blood cells gets stuck, it causes what is called a sickle cell crisis and this can cause a great deal of pain.

"Ezrah is also prone to serious infections.

"He takes penicillin every day as well as folic acid to boost his immunity.

"Id never given blood before having Ezrah.

"It wasnt until the first time doctors told me that they were going to have to transfuse him that I realised how important it was.

"Im pregnant now so I cant do it myself just yet, but as soon as I can sign up, I will.

"You never know whats round the corner.

"Its not until it happens to someone close to you that you realise how important it is."

'While recovering he's had more than 50 transfusions'

JACOB JESSEL lives with mum Emma Riley, 47, an NHS project manager, dad Nick Jessel, 44, a sales manager, and brother Sam, eight, near Grimsby, Lincs. Emma says:

"Jacob was diagnosed with a rare blood disorder when he was seven.

"We went on a camping trip and he was bitten by a mosquito. A huge bruise came out, which covered most of his forearm.

"Our GP took blood and told us his blood count was dangerously low and that we had to take him straight to hospital.

"It was a huge shock and it was obvious to us that doctors feared he had leukaemia.

"Jacob was given an emergency bone marrow biopsy at Sheffield Childrens Hospital and we were told he probably had cancer.

"Waiting for the results of the biopsy was horrible.

"We were relieved when the tests came back negative, but more tests revealed he had an incurable bone marrow disorder.

"Doctors said hed need a transplant, which he had in 2017.

"There was only one match on the register at the time so we went ahead with it. But sadly that didnt work.

"About a month later, he had a transplant using his dads stem cells, which has been effective.

"While recovering, he had more than 50 blood transfusions.

"He now attends a follow-up clinic every four to six months to make sure his blood keeps working properly.

"Before Jacob was ill, I was one of these people who never got round to giving blood.

"I thought it was a good thing to do but I kept putting it off.

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"But every time a unit of blood was delivered to the ward for Jacob, I felt incredibly relieved that someone, somewhere, had taken the time to give blood.

"Now I give blood regularly. Its a good feeling to know you are helping someone else.

"I know how grateful the recipient will 7 be. Its the best gift anyone can give."

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None of these four beaming children would be alive today if a stranger had not given blood - The Sun

Skin Stem Cells Comments Off on None of these four beaming children would be alive today if a stranger had not given blood – The Sun | January 2nd, 2020

In theglobal stem cells marketa sizeable proportion of companies are trying to garner investments from organizations based overseas. This is one of the strategies leveraged by them to grow their market share. Further, they are also forging partnerships with pharmaceutical organizations to up revenues.

In addition, companies in the global stem cells market are pouring money into expansion through multidisciplinary and multi-sector collaboration for large scale production of high quality pluripotent and differentiated cells. The market, at present, is characterized by a diverse product portfolio, which is expected to up competition, and eventually growth in the market.

Some of the key players operating in the global stem cells market are STEMCELL Technologies Inc., Astellas Pharma Inc., Cellular Engineering Technologies Inc., BioTime Inc., Takara Bio Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.

As per a report by Transparency Market Research, the global market for stem cells is expected to register a healthy CAGR of 13.8% during the period from 2017 to 2025 to become worth US$270.5 bn by 2025.

Request a Sample of Stem Cells Market Report

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Depending upon the type of products, the global stem cell market can be divided into adult stem cells, human embryonic stem cells, induced pluripotent stem cells, etc. Of them, the segment of adult stem cells accounts for a leading share in the market. This is because of their ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.

Depending upon geography, the key segments of the global stem cells market are North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. At present, North America dominates the market because of the substantial investments in the field, impressive economic growth, rising instances of target chronic diseases, and technological progress. As per the TMR report, the market in North America will likely retain its dominant share in the near future to become worth US$167.33 bn by 2025.

Investments in Research Drives Market

Constant thrust on research to broaden the utility scope of associated products is at the forefront of driving growth in the global stem cells market. Such research projects have generated various possibilities of different clinical applications of these cells, to usher in new treatments for diseases.Since cellular therapies are considered the next major step in transforming healthcare, companies are expanding their cellular therapy portfolio to include a range of ailments such as Parkinsons disease, type 1 diabetes, spinal cord injury, Alzheimers disease, etc.

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The growing prevalence of chronic diseases and increasing investments of pharmaceutical and biopharmaceutical companies in stem cell research are the key driving factors for the stem cells therapeutics market. The growing number of stem cell donors, improved stem cell banking facilities, and increasing research and development are other crucial factors serving to propel the market, explains the lead analyst of the report.

This review is based on the findings of a TMR report, titled, Stem Cells Market (Product Adult Stem Cell, Human Embryonic Stem Cell, and Induced Pluripotent Stem; Sources Autologous and Allogeneic; Application Regenerative Medicine and Drug Discovery and Development; End Users Therapeutic Companies, Cell and Tissues Banks, Tools and Reagent Companies, and Service Companies) Global Industry Analysis, Size, Share, Volume, Growth, Trends, and Forecast 20172025.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

ContactTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY 12207United StatesTel:+1-518-618-1030USA Canada Toll Free:866-552-3453Email:[emailprotected]Website:http://www.transparencymarketresearch.com

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Stem Cells Market in The Region Is Anticipated To Expand At a CAGR of 13.8% During the Period from 2017 to 2025 - Market Research Sheets

Spinal Cord Stem Cells Comments Off on Stem Cells Market in The Region Is Anticipated To Expand At a CAGR of 13.8% During the Period from 2017 to 2025 – Market Research Sheets | January 1st, 2020

Ryan Straschnitzki, a 20-year-old hockey player who was involved in the Humboldt Broncos bus crash is home after undergoing surgery in Thailand. Straschnitzki was paralyzed when the team bus hit a westbound semi-trailer truck on April 6, 2018. 16 people on the bus were killed due to the awful accident.

On Sunday night, Straschnitzki made his way into the Calgary airport from Thailand. He told Global News, It feels good. I mean I felt that cold, cold wind hit my legs, so Im feeling good. Its good to be back.

WE MADE ER back. 35 days away. Huge thanks to our peeps in Thailand and Air Canada. Cant wait for Family Christmas decorating. Brawls. Haha. Ryan with his therapy cat, Bronz (Named after Dana) and Dexter. Hugs were huge and long. pic.twitter.com/Ujshile6nd Strazsr (@strazsr) December 9, 2019

Straschnitzki was paralyzed from the chest down in the accident and had to have an epidural stimulator inserted into his spine along with having stem cells injected.

Straschnitzki is quoted saying, It was incredible. I mean the last time I walked beside my dad was before the accident and before I moved away. So doing that again and just seeing the look in his eyes is motivating to me.

His father, Tom, said, When I actually saw him move his leg, it just took me back to imagining his last steps going onto that bus on that fateful day. And I was just thinking maybe he can go back on the bus one day.

The surgery that Straschnitzki required is not yet approved by Health Canada, or covered by public insurance, so it can cost close to $100,000. Because of this, Straschnitzki and his family had to make the trip to Thailand, unhappy with the Canadian health-care system.

Our health-care system is kind of lacking in this area for spinal cord injuries and I think its huge that Thailand and some other places are getting this started. Said Straschnitzki hoping to help get the ball rolling on the issue.

Licenced Spinal Cord stimulators are given by Health Canada but are just for pain relief and not for the recovery of motor skills.

Just getting that feeling of being able to move something that I wasnt able to move before, and I know core is a huge part of my disability, so anything below my chest is crucial. And after the programming it really helped, said Straschnitzki.

He is planning to take some time to rest before getting back to the ice and physiotherapy. Straschnitzki is also optimistic about making the Canadian Olympic team for sledge hockey in the future.

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Husband gives wife, 74, the gift of his kidney - The Post Millennial

Spinal Cord Stem Cells Comments Off on Husband gives wife, 74, the gift of his kidney – The Post Millennial | January 1st, 2020

HUNTINGTON Serucell Corporation, a cosmeceutical company based in Huntington, has developed the worlds only dual-cell technology to create and produce anti-aging skincare products, and they did it in Huntington.

Serucell KFS Cellular Protein Complex Serum is made start to finish at Serucells laboratory on the south side of Huntington.

This has been one of the best kept secrets in West Virginia, said Cortland Bohacek, executive chairman and a co-founder of Serucell Corporation.

The company soft launch was in September 2018 at The Greenbrier Spas. The Official online launch was April 2019 and is getting exposure with some well known sellers like Neiman Marcus, local dermatologist and plastic surgeons offices and several other retail locations from New York to California. It is also sold online at serucell.com.

One person that has tried the product is Jennifer Wheeler, who is also a Huntington City Council member.

As a consumer I have an appreciation of the quality of the product and the results Ive seen using it, she said. It has been transformative for my skin and seems like its success will be transformative for our city as well.

She said Serucell and the people behind it are impressive on every level.

In my role on council, Im especially grateful for the companys conscious effort to stay and grow in our city, Wheeler said.

A one-ounce bottle of the serum costs $225. The recommended usage is twice per day and it will last on average of about six weeks.

Serucells active ingredient is called KFS (Keratinocyte Fibroblast Serum), which is made up of more than 1,500 naturally derived super proteins, collagens, peptides and signaling factors that support optimal communication within the cellular makeup of your skin.

This is the first and only dual-cell technology that optimizes hydration and harnesses the power of both keratinocytes and fibroblasts, two essential contributors to maintaining healthy skin by supporting natural rejuvenation of aging skin from the inside out, said Jennifer Hessel, president and CEO of the company.

When applied to the skin, KFS helps boost the skins natural ability to support new collagen and elastin, strengthen the connection and layer of support between the upper and lower layers of your skin. The result, over time is firmer, plumper and smoother skin, according to Hessel.

Why it works so naturally with your skin is because it is natural, Hessel said. These proteins play an important role in strengthening the bond between the layers of your skin, and thats where the re-boot happens.

KFS is the creation of Dr. Walter Neto, Serucells chief science officer and co-founder of the company. Neto is both a physician and a research scientist, specializing in the field of regenerative medicine with an emphasis on skin healing and repair.

Neto said Serucells technology unlocks the key to how our cells communicate and harnesses the signaling power actions to produce the thousands of bioactive proteins necessary to support the skins natural rejuvenation.

Originally from Brazil, Neto studied at Saint Matthews University and completed his clinical training in England. His clinical research on stem-cell cancer therapies, bone and tissue engineering and wound and burn healing led to his discovery in cell-to-cell communication, and ultimately the creation of Serucells KFS Cellular Protein Complex Serum.

Neto received multiple patents for the production method of Serucell KFS Serum. He lives in Huntington with his wife and four golden retrievers and works alongside his longtime friend, Dr. Brett Jarrell.

I have known Brett since I was 18 years old, Neto said.

Jarrell practices emergency medicine in Ashland, Kentucky, and oversees all aspects of quality control for Serucell. He received his bachelors degree in biology from Wittenberg University, his masters degree in biology from Marshall University and his medical degree from the Marshall University School of Medicine. Jarrell completed his residency at West Virginia University and is board certified by the American Board of Emergency Medicine.

Jarrell has served as a clinical instructor of emergency medicine at the Marshall School of Medicine, president of the West Virginia chapter of the American College of Emergency Medicine and he has published a number of peer-reviewed journal articles on stroke research.

Jarrell also lives in Huntington.

Another co-founder of the company is Dr. Tom McClellan.

McClellan is Serucells chief medical officer and director of research and is a well-respected plastic and reconstructive surgeon with a private practice, McClellan Plastic Surgery, in Morgantown.

McClellan completed his plastic and reconstructive surgery training at the world-renowned Lahey Clinic Foundation, a Harvard Medical School and Tufts Medical School affiliate in Boston, Massachusetts. While in Boston, he worked at Lahey Medical Center, Brigham and Womens Hospital, as well as at the Boston Childrens Hospital. McClellan is board certified by the American Board of Plastic Surgery.

In addition to his practice and role at Serucell, McClellan utilizes his surgical skills through pro bono work with InterplastWV, a non-profit group that provides comprehensive reconstructive surgery to the developing world. He has participated in surgical missions to Haiti, Peru and the Bahamas.

McClellan lives in Morgantown with his family.

All three doctors here have strong connections to West Virginia, and we didnt want to leave, Neto said. We all want to give back to West Virginia, so that is the main reason we have our business here in Huntington.

We are building a company we believe can make a difference in the community, Hessel added. Our goal is to grow Serucell and build our brand right here in Huntington. There is a pool of untapped talent here in Huntington. When we expand our business here, we can provide another reason for young people to be able to stay and grow their careers, whether it is in science, operations or manufacturing. The team is a pretty excited to make an impact in the community where it all started.

Hessel decline to give sales numbers, but said the business has been growing each year since the product was introduced. She also declined to give the number of employees at the facility, but did say it has sales representatives across the country.

For more information, visit serucell.com.

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Firm adds a new wrinkle to anti-aging products - The Logan Banner

Skin Stem Cells Comments Off on Firm adds a new wrinkle to anti-aging products – The Logan Banner | January 1st, 2020

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

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Gene Therapies Make it to Clinical Trials - Discover Magazine

Bone Marrow Stem Cells Comments Off on Gene Therapies Make it to Clinical Trials – Discover Magazine | December 31st, 2019

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy. "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency." Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors." The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured. "This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting." In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden. For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs. The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself. "It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy. Scientists practising the technique insert a normal gene into cells containing a defective gene. It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient. Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union. They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster." Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period." "We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies. They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor. However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine. Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans. The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process. "That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations. "It's very easy to do if you don't care about the consequences," Musunuru added. Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability. There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US. The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online. "Not everyone is a biologist or scientist," she said. And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops? Charpentier thinks that technology generally tends to be used for the better. "I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18

Bone Marrow Stem Cells Comments Off on Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research – News18 | December 31st, 2019

The 2010s are coming to an end, and looking back there have been some pretty amazing advances and innovations in health and science.

Advances in prosthetic limbs

Prosthetic limbs have been around since ancient times. In Egypt, a prosthetic wooden toe was found on a mummy dating back 3,000 years. By the Dark Ages, inventors could incorporate hinges on prosthetic arms used by knights. In modern times, the field of prosthetics has turned to incorporating more technology into physical stand-ins for limbs. In the last several years, theres been a boom in advances that have led to the best and most useful prosthetics weve ever seen.

Reports from the early 2010s talked about the potential for new technology to allow people to control prosthetics with their minds and to receive sensory information from their devices. It may have been a reach in the early part of the decade, but now it is literally within grasp. There are new prosthetic hands being tested that give the user the ability to grab objects with their thoughts and even to sense the texture of what they are touching. New bionic hands allow the user to feel again by sending signals back to the brain about the things they are touching, like whether its hard or soft. Other research groups have been working on bionic arms that can move based on the users thoughts through a brain-computer interface. While these have demonstrated its possible to accomplish these goals in the lab, theres still more to be done before people can use these devices outside in the real world.

Many of these advanced prosthetics are still prototypes and may not reach the general population for a while. Luckily, cheaper 3D printers have made simple prosthetics more accessible. These are important because a prosthetic device can improve the quality of life for people. For example, this person has been printing prosthetic hands and arms for people in Africa after watching an online tutorial. New materials that go into 3D printers are cheaper than they used to be and are being used in prosthetics to provide a more affordable option for patients.

Although prosthetics have been around for ages in some form or another, they arent always used. One variable to consider is the social acceptance of having a prosthetic. Theres still a lot of stigma around disabilities and many people may reject prosthetics even if they are available. In 2012, an athlete with both feet amputated competed in the mens 400 meter race at the Olympics in London. There was some controversy over whether the runner with a prosthetic foot should be allowed to run in races with people who dont have prosthetics or if they should only be allowed in competitions specifically for people who have them. Prosthetics also need to be comfortable and usable in order to be successfully adopted. In one study, about 4.5 percent of people rejected prosthetics and 13.4 percent stopped using their prosthetics. As the new prosthetics that are more natural and intuitive to use come to market, hopefully more people will benefit, and the social barriers to acceptance will disappear.

CRISPR

The genome modification technique called Clustered Regularly Interspaced Short Palindromic Repeats, aka CRISPR, was a culmination of a few decades of work by scientists, and major studies explaining the method were published in 2013. The version of it called CRISPR-associated protein 9 or CRIPSR-Cas9 is what most researchers are specifically using in most cases. It involves a regular gene editing mechanism that happens in bacteria. The bacteria can take sections of DNA from attacking viruses and essentially use that to remember the viruses if they return. When the virus is back, the bacteria can target the matching sections of DNA in the virus, cut it and disable the virus.

Though 2013 was only six years ago, as far as science goes, CRISPR has been moving at lightning speed towards practical applications. Using CRISPR to edit a gene sequence, researchers can now add, delete or modify DNA segments more quickly and accurately than ever before. Since the technique was developed, researchers have used CRISPR to target diseases caused by a single gene like cystic fibrosis or sickle cell disease.

Probably the most infamous use of CRISPR are the CRISPR babies. In late 2018, a Chinese researcher, He Jiankui, claimed to have used CRISPR to modify the genomes of two babies to include a mutated version of a gene that protects against HIV. This case was and is highly controversial for the ethical concerns with genetically modifying a human genome at the embryo level, or germline, meaning it can be passed down to future generations and has not been done before in humans. Recently, MIT Technology Review obtained excerpts from Hes research, and experts say that the report and data may be untrustworthy. This means it is still unclear if He and collaborators actually successfully modified the babies genomes. The scientific community overall condemns this way of using CRISPR to edit a human germline genome and has called for an international moratorium on it until a framework can be agreed on.The researcher has been sentenced to three years in prison in Shenzhen, China.

As fraught with controversy as the CRISPR babies may be, CRISPR technology still holds a lot of promise and can be used responsibly, supporters say. For example, researchers are using it to target cancer cells by taking a patients immune cells, modifying them using CRISPR and then infusing the patient with the modified cells. For blood diseases, a patient with sickle cell disease is reported to be responding well to a CRISPR treatment that has allowed her body to produce a crucial protein.

Another area that has boomed this decade partly because of CRISPR technology is stem cell therapy, which well get into in the next section.

Stem cell therapy

Technically, the only Federal Drug Administration (FDA)-approved stem cell therapies are blood-forming stem cells derived from umbilical cord blood. Blood-forming stem cells are used to treat patients with cancer after chemotherapy has depleted blood cells, as well as patients with blood disorders like leukemia whose bone marrow tissues are damaged. These types of treatments have been around for about 30 years, but in the 2010s weve seen potential for more uses of stem cells in health care.

The main idea behind stem cell therapy is that because the cells are pluripotent meaning they can become many other types of cells they can be introduced into parts of the body that are damaged and need new cells. On top of that, researchers can now extract some types of stem cells from a persons body, so no need for umbilical cords. This opens up the possibilities for highly personalized treatment where one person can be treated with stem cells from their own body.

Researchers are exploring how stem cells can be used to treat liver disease, cerebral palsy, stroke, brain injury and others. There are many ongoing research-backed clinical trials for stem cell therapy. A quick search for stem cell therapy on the governments clinical trial database turns up 5,638 results. And because of the work necessary to even get to the clinical trial stage, theres likely an order of magnitude more stem cell therapy studies in the pre-clinical trial stages.

Stem cell therapy is also being offered in for-profit clinics around the U.S. In these cases, the clinics are typically taking fat tissue from a patient, isolating the stem cells and then administering the stem cells back to the patient. In some cases, the treatments may lead to health complications, like blindness in a few extreme cases, and the FDA warns that such treatments are unapproved and potentially harmful. The FDA is ramping up regulation of stem cell clinics and earlier this year took a specific clinic in Florida to court.

Although there are many stem cell clinics offering unproven stem cell therapies, its not all hype. Granted that its difficult to pass the clinical trial stage to get FDA approval, stem cell research may lead to new treatments for several health conditions that could completely change the health care landscape.

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The 3 most important health innovations of the past decade - The Hill

Bone Marrow Stem Cells Comments Off on The 3 most important health innovations of the past decade – The Hill | December 31st, 2019

Doctor close-up of a doctor showing a picture of a kidney on a tablet in a hospital

With a float in this years Rose Bowl parade celebrating organ donation, there are a lot of questions many have about the process and why they should donate their organs.

Legacy of Hope, the Alabama organ donation alliance, said over 1,400 Alabama residents are waiting for a life-saving transplant, with 471 lives saved in 2018.

2.9 million residents across the state are on the registry.

Can I become an organ donor?

The federal government organ donation website, Organdonor.gov, says anyone 18 and older can join the national and state organ donor registries and donate as long as they and their organs are in healthy condition.

The Tennessee donor registry also allows anyone between 13 and 17 to join as long as they have a state ID, drivers license, or leaners permit. However, their parents will have the final say on organ and tissue donation if that decision needs to be made.

Even if you have health issues, you could still donate even one organ, which could save or improve a life.

What can be donated?

How do I register to donate?

There are two registries: The National Donor Registry and the state registry.

In Alabama and Tennessee, if you checked yes to organ donation when applying for or renewing your license, youre already on the state list.

If you didnt check yes, you can make your decision when applying for or renewing your drivers license or state ID at your local DMV or visit your states registry online.

In Alabama, Legacy of Hope manages the state registry, and you can sign up here.

In Tennessee, Donate Life Tennessee manages the state registry donation registry, and you can sign up here.

Youll need to check yes every time you renew to stay on the list.

You can join the national registry hereor in the iPhone Health app.

Who will get my organs if I decide to donate?

Its possible anybody could get your organs if you donate. People of different races match frequently, according to organdonor.gov.

The matching process includes many factors such as location, how long a recipient has been on the list, medical need, and determining blood and tissue type.

The Organ Procurement and Transplantation Network handles the matching process and it varies based on the organ being transplanted.

Does my decision to donate affect the care I get in the hospital?

No. The medical teams saving your life will do everything in their power before donation becomes a possibility. A separate team handles organ retrieval should it be necessary.

The donation process only begins once brain death is confirmed. In those cases, a potential donor must have no brain activity and be unable to breathe without a machine.

Legacy of Hope says in Alabama, two doctors have to mutually agree that a patient is brain dead before the process starts.

Where can I find more information?

If youre trying to decide or just want more information, there are multiple resources.

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Curious about organ donation? Heres what you need to know - WHNT News 19

Bone Marrow Stem Cells Comments Off on Curious about organ donation? Heres what you need to know – WHNT News 19 | December 31st, 2019

Soft tissue injuries in muscles, tendons and ligaments, andosteoarthritis, can make moving around painful and limit your physical activity. ButDr. James Presley,a Mayo Clinic physical medicine specialist, says two specialized treatments are growing more common and can help you heal faster.

Platelet-rich plasmais a specialized treatment that Dr. Presley says can bring relief for many patients dealing with soft tissue injuries.

Platelet-rich plasma is a way of trying to harness the bodys immune system or the bodys own ability to heal tissues, Dr. Presley says. [We] concentrate it and then spot-shoot it into the area of injury.

Dr. Presley says the process involves taking blood from your arm, processing it to concentrate the platelets, then injecting it directly into the affected area.

These treatments seem to be helpful in helping the healing process move along when it comes to tendon and ligament injuries, and potentially to help decrease pain and improve function in a joint that has some arthritis, Dr. Presley says.

The second treatment is calledbone marrow aspirate concentrateand involves extracting cells, including stem cells, from bone marrow in the pelvis; processing them into a solution; and injecting them into a painful joint.

The studies that have been done with this have shown patients have decreased pain and thereby improved function of a joint with mild to moderate osteoarthritis, Dr. Presley says.

But he says the best thing you can do is protect your muscles, tendons, and ligaments from injury by finding a happy medium between staying active and avoiding overuse.

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Advances In Treatment Of Soft Tissue Injuries (Video) - South Florida Reporter

Bone Marrow Stem Cells Comments Off on Advances In Treatment Of Soft Tissue Injuries (Video) – South Florida Reporter | December 31st, 2019

NewsScienceIt's likely to be an eventful year for gene editing and stem cell research

Tuesday, 31st December 2019, 7:03 am

Gene editing

It was suggested that prime editing has the potential to mend about 89 per cent of the 75,000 harmful genetic mutations that lie behind hereditary diseases, such as cystic fibrosis and the blood disorder sickle cell disease.

3D rockets

Major strides were made in rocket science last year, with a number of 3D-printed engine prototypes being successfully tested.

This year, Relativity Space, a California startup, hopes to go one better. It plans to become the first company in the world to print almost an entire rocket 95 per cent of it which it hopes will be ready for launching at the end of the year.

Only a handful of components, such as electronics and circuit boards, will have to be made by hand for the craft, named Teran 1.

If successful, the launch will pave the way for numerous 3D-printed rockets to be sent into space much more quickly and cheaply than they are at the moment.

Stem cells

Scientists are working around the world on trials of promising stem-cell treatments for blindness, spinal cord injury, heart failure, diabetes, Parkinsons disease and lung cancer, and some of the first results should become available later in the year.

Embryonic, or pluripotent, stem cells have extraordinary medical potential because they can develop into any of the 220 or so mature, specialised cells of the body, from insulin-making pancreatic cells to the nerve cells of the brain.

Mars

The 2020 mission of the ExoMars programme, if all goes to plan, will deliver a European rover and a Russian platform to the surface of Mars.

ExoMars will be the first mission to combine the capability to move across the surface of the planet and to the ability study Mars at depth. Meanwhile, Nasa will launch a separate mission to study the habitability of Mars and prepare for future human missions.

Smart needle

They have demonstrated that the technique works in the laboratory, and are in the early stages of a three-year clinical trial to test it in living people.

The researchers have focused on lymphoma so far, but said that they are hopeful the technique could also be used further down the line to diagnose other forms of the disease, such as breast and prostate cancer.

At the moment, diagnosing lymphoma can be an invasive process that involves a surgical biopsy followed by a nerve-racking wait for the result, which can often take two weeks or more.

The smart needle uses light to pinpoint cancerous tissues almost instantaneously.

Using a technique called Raman spectroscopy, the optical biopsy measures the light scattered by tissues when a laser contained in the needle is shone on it.

The light scatters differently from healthy tissues than it does from diseased tissues, meaning that doctors can make their diagnosis straight away.

Japan's robotic Olympics

Japan has pledged to make the 2020 Tokyo Olympics and Paralympics the most innovative in history by deploying robots to assist spectators and staff during the games.

The Human Support Robot (HSR) and Delivery Support Robot (DSR), developed by Toyota, will be used in tandem.

HSR, a one-armed robot about 3ft (1m) tall, can hold objects, pick things up off the ground and reach up high. It can move by itself, or can be controlled remotely as it attends to people in wheelchairs, guiding them to their seats and helping to carry items.

When people order food or drinks using a tablet computer, DSR will transport the items in a basket and HSR will then deliver them directly to guests.

Waste to Energy

The worlds largest waste-to-energy plant is set to open on the outskirts of Shenzhen, China. The new plant is made to handle 5000 tons of waste per day, burning the waste to generate electricity.

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Predicting shifts in technology and science can be tricky, but here's what could happen in 2020 - inews

Spinal Cord Stem Cells Comments Off on Predicting shifts in technology and science can be tricky, but here’s what could happen in 2020 – inews | December 31st, 2019

This undated screen grab shows the cell-division of two fertilized human embryos during the first 24 hours of embryonic development following IVF treatment at a private clinic in London. ( Jim Dyson/Getty Images )

LOS ANGELES - Some of the scientific advancements of the 2010s have been truly mind-blowing, and perhaps none more so than the leaps and bounds weve made in the realm of reproduction.

This was not only the decade in which the first three-parent baby was born, it was the era when a rogue scientist chose to make edits to a set of twin girls DNA, making real the long-imagined scenario of genetically altering human beings while simultaneously thrusting the deeply complicated ethical discussions surrounding this practice into the limelight.

These are the five most life-altering breakthroughs in reproduction from the past decade.

In 2018, Chinese biophysics researcher He Jiankui announced that he had used the gene-editing tool CRISPR to modify the genes of two twin girls before birth. He and his team said that their goal was to make the girls immune to infection by HIV through the elimination of a gene called CCR5.

When the news broke, many mainstream scientists criticized the attempt, calling it too unsafe to try. Where some people saw the potential for a new kind of medical treatment capable of eradicating genetic disease, others saw a window into a dystopian future filled with designer babies and framed by a new kind of eugenics.

At the time, Dr. Kiran Musunuru, a University of Pennsylvania gene-editing expert, said Hes work was unconscionable... an experiment on human beings that is not morally or ethically defensible.

Other experts believe Hes work could propel the field of gene editing forward.

The twins, known as Lulu and Nana, have continued to make headlines since their birth. The gene modification that He claims to have carried out may have caused some unintended mutations in other parts of the genome, which could have unpredictable consequences for their health long term something many scientists who argue against Hes work cite as a reason to hold off on using gene-editing technology on humans.

Only time will tell what will happen to Lulu and Nana and if the edits to their DNA ultimately help or hurt them, but their story pushed the topic of human gene-editing and the ethics surrounding it to the forefront of the global scientific community.

In 2016, a technique called mitochondrial transfer was used successfully for the first time to create a three-parent baby grown from a fathers sperm, a mothers cell nucleus and a third donors egg that had the nucleus removed.

This technique was developed to prevent the transmission of certain genetic disorders through the mothers mitochondria. The majority of a three-parent babys DNA would come from his parents in the form of nuclear DNA, and only a small portion would come from the donor in the form of mitochondrial DNA.

A team led by physician John Zhang at the New Hope Fertility Center in New York City facilitated the birth of the first three-parent baby in April 2016.

Using human pluripotent stem cells, researchers were able to make the precursors of human sperm or eggs. In other words, they reprogrammed skin and blood stem cells to become an early-state version of what would eventually become either sperm or an egg.

"The creation of primordial germ cells is one of the earliest events during early mammalian development," Dr. Naoko Irie, first author of the paper from the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge told Science Daily. "It's a stage we've managed to recreate using stem cells from mice and rats, but until now few researches have done this systematically using human stem cells. It has highlighted important differences between embryo development in humans and rodents that may mean findings in mice and rats may not be directly extrapolated to humans."

A 2018 study showed that gene editing can allow two same-sex mice to conceive pups, and two female mice were able to successfully create healthy pups that then went on to reproduce themselves.

A team of researchers at the Chinese Academy of Sciences in Beijing, led by developmental biologist Qi Zhou, were able to use gene editing to produce 29 living mice from two females, seven of which went on to have their own pups. They were able to produce 12 pups from two male parents, but those offspring were not able to live more than two days.Whether or not the method can one day be used in same-sex human reproduction is still up for debate.

For the first time ever, Chinese scientists were able to clone two primates using the technique that produced Dolly the sheep, the first mammal to be cloned from an adult somatic cell via nuclear transfer.

The two cloned female macaques were named Zhong Zhong and Hua Hua, and their successful birth opened up the possibility of using the same cloning method to one day clone humans.

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What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it - FOX 5 Atlanta

Skin Stem Cells Comments Off on What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it – FOX 5 Atlanta | December 30th, 2019

For Alexander Fleming, leaving a petri dish out in the air led to his now famous discovery of antibiotics. For Madeline Lancaster, leaving stem cells in a shaker led to the discovery of a new model for neuroscience: brain organoids. These blobs of tissue, grown from human stem cells, resemble some of the essential parts of the human brain. Although they are as small as apple seeds, brain organoids may hold the key to understanding one of lifes great mysteries: the human brain.

Our brain is, arguably, the organ that most makes humans what we are. Our cerebral cortex, the outermost layer of the brain, underpins human cognition. When things go wrong in the cerebral cortex, either as we develop or as we age, this can cause neurological or psychiatric diseases. Neuroscientists have been trying to understand brain development and disease, but they have run into a pretty basic problem: We (usually) cannot collect brain tissue from living people. So neuroscientists arelimitedto studying tissue that is donated by those who have died or observing a living brain behave in an MRI.

To help fill in the gaps in our knowledge of the brain, scientists have turned to the proverbial lab rats (which are actually mice). Mice, rats, primates, and other animals have given scientists the chance to tease apart the roles of genes, molecules, and cells in brain development and disease. Some important insights were gained from these animals. Genetically engineered mice helped researchers understand how a protein called alpha-synucleincan misfold and clump together in theParkinsons-diseased brain, potentially injuring nerve cells. Research intoAlzheimers disease got a boost by mice that were genetically engineered to have mutations linked to Alzheimers. These mice have helped scientists understand how misfolded beta-amyloid proteins stick together in plaques in the brain, a hallmark of the disease.

But a mouse is not a human: mice do not behave as humans do; mouse brains are simpler than human brains; and the mouse genome is not the human genome. Researchers argue that modified mice or other animals do not reflect the complexity of humans, let alone the complexity of neurological or psychiatric conditions. For many conditions, researchers do not even know which gene defects are part of the underlying cause. Instead of creating a mouse that has the same gene defect as that found in human patients, researchers have had to make do with animal models that behave similarly to humans.

In one test for depression-like behavior, researchers hold mice upside down by their tail and measure how long they struggle against it. Mice that give up sooner are judged to show greater despair. But researchers are rightly skeptical. We can make models by challenging mice in different ways and looking at their behavior but its not at all clear that these animals have the same disease that we do, the neuroscientist Fred H. Gage, President of the Salk Institute for Biological Studies,said.

What then? Four millimeter brain organoids might seem an unlikely source for finding therapeutic breakthroughs for complex diseases. But much hope has been put into them since Madeline Lancasterpresented the first such minibrains in 2013. As with many scientific developments, her discovery had an element of serendipity, but cant be reduced to it.

Lancaster, then a postdoc in a Vienna laboratory, wanted to understand how developing brain cells switch from dividing, when they make more of themselves, to differentiation, when they turn into neurons or glia cells. To start off, Lancaster used techniques to coax stem cells, which can develop into pretty much any tissue, towards becoming neurons. But she had also been intrigued by the success of another research team, which grew mini-guts in Matrigel, a gelatinous protein mixture. So once Lancaster had coaxed the stem cells into becoming neural cells, she took clusters of them and put them into a drop of Matrigel. This gave the cells enough support to grow into larger and more complex structures.

Gently shaking the cells in a bioreactor, caused them to specialize into recognizable rudiments of the human brain. Within about two months, the brain organoids had grown structures similar to those found in the human brain, including the cerebral cortex, the seat of human cognition. Organoids also havegenetic similaritieswith the developing human brain. Moreover, many of the neurons in the organoids fired off electrical signals, the messages with which brain cells communicate.

Lancaster immediately realized one of the big promises of brain organoids: As stem cells are their starting material, researchers can take skin samples from adults and re-program those cells into stem cells. These then provide material for a personalized brain organoid. In their first presentation of organoids, the researchers grew personalized organoids from the skin cells of a person with microcephaly. Microcephaly, a condition where the brain is smaller than normal, is difficult to study using mouse models. The researchers took a step toward figuring out why neuron-producing cells stop their job too soon, which could ultimately result in too few neurons. When they added a copy of the faulty gene, the researchers grew organoids with more neuron-producing cells and, ultimately, more nerves.

Brain organoids certainly have their limitations: No two organoids are the same, potentially obscuring differences between personalized organoids. A lack of blood supply keeps the organoids small, as it limits the amount of oxygen that can get into their center. With about a couple of million neurons, a brain organoid has twice as many neurons as a cockroach, but far fewer than an adult zebrafish. Nevertheless, organoids have been used to investigate schizophrenia and autism, and scientists hope to use them to study a range of disorders, from Parkinsons to Alzheimers to eye conditions, like macular degeneration.

With time and intensive research, brain organoids are now better understood and being used in more complex experiments.One studyfound that organoids left to develop for 8 months formed neuronal circuits that sparked with activity, and grew light-sensitive neurons that responded when light was shone on them. Another lab has developedorganoids that produce brain waves similar to those of premature human babies.

Other researchers have developed workarounds to overcome the lack of blood supply. Inanother study, led by Fred Gage, neuroscientists transplanted human brain organoids straight into mouse brains. The organoids connected with the mouses blood supply, and connections between the human organoid and the animals brain sprouted.

This brings up ethical questions: the small blobs of brain tissue arent fully fledged brains, sitting in vats thinking about the meaning of life. The brain waves observed in some mature organoids alone are unlikely to be enough to produce complex brain functions. And, isolated from sensory input,it is unclear whether the organoids could even learn cognitive processes.

But implanting brain organoids into the brain of an actual living mouse could link the blob with the animals senses and motor system. These experiments hark to a related debate raging in science, about the creation and use of chimeras (animals into which human cells have been implanted). While in the US, the National Institutes of Health put in place a moratorium on funding research that investigates animal embryos containing human cells in 2015, in March 2019, Japanannounced a reversal of its ban, allowing scientists to grow human cells in animal embryos that are carried to term.

Arecent perspectivepublished inCellposited that, at the moment, the question isnt whether we humanize an animal into which a human brain organoid is implanted. Instead, it is important to ask whether the organoid enhances specific brain functions in the chimera, and at what point this enhancement crosses the line, becoming harmful and unethical. The authors argue that current studies are more likely to worsen brain function than to improve it.

At the moment, researchers need to make a surgical cavity to accommodate the organoid, which likely harms brain function. Once organoids can make up this deficit, which would be a notable achievement from a clinical perspective, and brain functions are enhanced above a critical threshold, perhaps the chimera should be given a higher moral status. This could go as far as giving the chimera the right of self-determination, the authors argue. Where this critical threshold lies is left open for debate, but the mirror test could be used to test for self-awareness in animals after organoid transplantation.

With brain organoids, the scientific community could be in danger of crossing yet another ethical line, some researchers warn. At this years meeting of the Society for Neuroscience, the largest annual meeting of neuroscientists, a group of scientists sounded the warning bell that research is coming close to creating sentient brain blobs in the lab, while some may have done so already.

The question here is at what point organoids, all on their own, develop consciousness or experience sentiments like pain. In 2018, a group of scientists, lawyers, ethicists, and philosophers, writing inNature,advocated for an ethical debate on brain organoids. With their initiative, they wanted to get ahead of the science, establishing guidelines before brain organoid research could raise immediate concerns.

At the meeting of the Society for Neuroscience, Elan Ohayon, director and founder of the Green Neuroscience Laboratory in San Diego, and his colleagues argued that serious concerns already have become reality. Ohayon presented a computer model which he believes helps to pinpoint when sentience is likely to arise. He suggests that some of the activity seen in organoids is reminiscent of the activity seen in developing animals and thatorganoid cultures may be capable of supporting sentient activity and behavior. Ohayon calls for a set of criteria to be applied to organoids that could help determine sentience and set ethical rules.

Brain organoids likely have a long way to go until they develop consciousness. And there is also likely a long way to go until they help researchers achieve therapeutic benefits. However, the promise of these blobs is so great that giving up brain organoid research altogethergiven the suffering caused by neurological and psychiatric diseases, and the lack of other modelscould itself be unethical. Whether or not any ethical lines have been crossed already, it is high time that neuroscientists, and society, come to terms with the question: What will organoids be able to tell us, and are we prepared to pay the price?

This article originally appeared on JSTOR Daily. Read the original here.

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The ethics of lab-grown "minibrains" - Quartz

Skin Stem Cells Comments Off on The ethics of lab-grown "minibrains" – Quartz | December 30th, 2019

Cardiac rhythm management refers to a process of monitoring functioning of the heart through devices. Cardiac rhythm management devices are used to provide therapeutic solutions to patients suffering from cardiac disorders such as cardiac arrhythmias, heart failure, and cardiac arrests. Cardiac disorders lead to irregular heartbeat. Technological advancements and rise in the number of deaths due to increasing incidences of heart diseases and increasing aging population are some of the major factors driving the cardiac rhythm management market. Heart disease is one of the primary causes of death in the U. S. Excess of alcohol consumption; smoking, high cholesterol levels, and obesity are some of the major causes of heart diseases.

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Cardiac rhythm management is conducted through two major devices: implantable cardiac rhythm devices and pacemakers. Implantable cardiac rhythm devices treat patients with an improper heartbeat. Based on the device, the cardiac rhythm management market can be segmented into defibrillators, pacemakers, cardiac resynchronization therapy devices, implantable defibrillators, and external defibrillators. Pacemakers are used to treat patients with a slow heartbeat. Based on the end user, the cardiac rhythm management market can be segmented into hospitals, home/ambulatory, and others.

North America has the largest market for cardiac rhythm management due to improved healthcare infrastructure, government initiatives, rise in incidences of cardiac disorders, growing number of deaths due to cardiovascular diseases,and increasing healthcare expenditure in the region. The North America market for cardiac rhythm management is followed by Europe. Asia is expected to witness high growth rate in the cardiac rhythm management market in the next few years due to increasing incidences of cardiovascular diseases, growing disposable income, rise in awareness regarding heart disorders and relevant treatments, and improving healthcare infrastructure in the region.

Increasing the prevalence of cardiovascular diseases, technological advancements, rise in life expectancy, increasing awareness regarding cardiac disorders, and government initiatives are some of the major factors that are expected to drive the market for cardiac rhythm management. In addition, factors such as a rise in disposable income, increasing aging population, and high cost associated with heart disease treatment are expected to drive the market for cardiac rhythm management. However, economic downturn, reimbursement issues, the importance of biologics and stem cells, and inappropriate use of the devices are some of the factors restraining the growth of the global cardiac rhythm management market.

Growing population and economies in the developing countries such as India and China are expected to drive the growth of the cardiac rhythm management market in Asia. In addition,factors such as innovations along with technological advancements such as miniaturization, introduction of MRI pacemakers, biocompatible materials and durable batteries, and continuous rise in aging population and increasing cardiovascular diseases such as arrhythmias, stroke, and high blood pressure are expected to create new opportunities for the global cardiac rhythm management market. An increasing number of mergers and acquisitions, rise in the number of collaborations and partnerships, and new product launches are some of the latest trends in the global cardiac rhythm management market. Some of the major companies operating in the global cardiac rhythm management market are

Other companies with significant presence in the global cardiac rhythm management market include

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Key geographies evaluated in this report are:

Key features of this report

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Cardiac Rhythm Management Market Development, Key Opportunity and Analysis of Leading Players to 2015 To 2021 - Info Street Wire

Cardiac Stem Cells Comments Off on Cardiac Rhythm Management Market Development, Key Opportunity and Analysis of Leading Players to 2015 To 2021 – Info Street Wire | December 30th, 2019

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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Stem Cell Assay Market Expected to Witness a Sustainable Growth over 2025 - Filmi Baba

Cardiac Stem Cells Comments Off on Stem Cell Assay Market Expected to Witness a Sustainable Growth over 2025 – Filmi Baba | December 30th, 2019

A former neurosurgeon, who worked with the UKs National Health Service, has a surprising prediction for the next decade. He claims human head transplants will become a reality by 2030.

Dr Bruce Matthew said this during an interview with The Telegraph on December 21. He says the revelation came to him while he was working on a science-fiction novel with futurist author Michal J Lee.

Ifyou transplant the brain and keep the brain and spinal cord together itsactually not impossible. The spinal cord is the most profound thing imaginable.You need to keep the brain connected to the spinal cord. The idea that you cut the spinal cord isutterly ridiculous, he told The Telegraph.

Previous(unsuccessful) attempts at the controversial procedure focused on severing thespinal cord from the brain and then transplanting the head after connectingnerves, blood vessels and meninges (the covering of the brain and spinal cord).

This happened in2017 on a human corpse. Controversial Italian neurosurgeon Dr Sergio Canavero and DrXiaoping Ren of Harbin Medical University, China carried out the 18-hour procedurewhich was later slammed by scientists and bioethicists the world over.

Dr Matthew admits that the future process will be tricky because shifting the spinal cord intact is impossible.

It will take a number of advancements and incremental steps but it will probably happen in the next 10 years, said the former surgeon, who was a clinical lead for neurosurgery at Hull University Teaching Hospitals NHS Trust in the UK and has 25 years of experience.

Hesays the feat will be accomplished with the help of cryogenics (freezing deadbodies in nitrogen), robotics, stem cell therapy and artificial intelligence.

Who will head transplants help?

People with terminal illnesses whose brains are still intact, those with neurodegenerative muscle diseases, the rich who have already frozen their bodies it costs somewhere between $28,000 and $200,000 in hopes of reincarnation.

Basically,those with an intact brain.

Why are head transplants so controversial?

Apartfrom the medical limitations and lack of scientific research on the topic, thebiggest issue that arises is an ethical one.

Doesthe person remain the same person? Will identity also be transferred? DrMatthew says shifting the spinal cord means shifting a persons consciousness. Asfor DNA, hes proposed shifting stem cells from the patient to the donors bodyso a new colony of original DNA can be built.

Scientists also say the donor body runs the risk of being paralysed because of the procedure. Bioethicists say the surgery will have profound psychological, legal and moral complications.

Follow SAMAA English onFacebook,Twitter,andInstagram.

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Transplanting human heads on to another body possible by 2030 - Samaa News

Spinal Cord Stem Cells Comments Off on Transplanting human heads on to another body possible by 2030 – Samaa News | December 29th, 2019

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

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2019: The year gene therapy came of age - INQUIRER.net

Bone Marrow Stem Cells Comments Off on 2019: The year gene therapy came of age – INQUIRER.net | December 29th, 2019

A mum whose ex bludgeoned her teenage son to death with a hammer before killing himself is making a heartfelt plea to strangers to try and save her own life.

Tania Morris fears this Christmas may be her last after being diagnosed with Hodgkin's Lymphoma shortly after the tragedy in Burslem, Stoke-on-Trent.

The 49-year-old, who was the only survivor of the brutal attack, was later told that her only chance of survival is a stem cell transplant, StokeonTrentLive revealed.

In the most cruel twists of fate, her only hope would have been heronly child, 19-year-old Nathan Bates, who was murdered just weeks before she was diagnosed with the deadly blood disease.

Tania and her loved-ones are now desperately urging people to join the Antony Nolan register in the hope they can get their very own Christmas miracle.

Tests on other family members including Tania's mum Viv, 69, and dad Robert, aged 70, showed they are not suitable as they are too old and one has a pacemaker.

And her brother Darren Morris, 48, was not a match either.

Meanwhile, her younger brother Adam Morris, who could have fitted the bill, died of a heart attack at the age of 41 four years ago.

Brave Tania, of Burlsem. told StokeonTrentLive: "It's heartbreaking. The doctors just keep saying we need a fit-and-healthy 19-year-old and that breaks my heart as that's how old Nathan was when he was murdered.

"My younger brother could have been a match but he died of a heart attack.

"My other brother Darren was devastated when he was tested and wasn't a match. He wanted so much to help me.

"Dad's only a half match. He's too poorly himself to go ahead but if and when it becomes life or death he could be a last roesort. They're worried it would kill both me and him.

"It would really be desperate measures if it comes to that. It would be my last option because it could kill me.

"My mum's not even been tested because she's had a heart bypass.

"We are just hoping that someone comes forward. It's my only chance of beating this."

Tania's ex, Robert Goodwin, attacked Nathan as he slept at his home, knowing Nathans mum would be out.

An inquest heard Goodwin then left the property and hanged himself in nearby woodland at a spot where Tania would routinely walk her dogs.

It is believed he did so with the intentions that his ex would discover not just her sons body, but also his.

At the time of Nathan's murder, Goodwin was on bail after being charged with assaulting Tania and had retained his liberty despite having breached a condition not to contact her.

And it was while she was coping with the stress of organising Nathan's funeral the following December that she first noticed her own health decline.

The official blood cancer diagnosis then came in January this year.

She has since endured endless rounds of chemotherapy - but has just been told the latest treatment is not working.

Tania said: "He's just a coward for what he did to Nathan. He wouldn't accept any responsibility for what he did. He wanted me to drop the charges but I refused because I was scared he would do it to someone else.

"He just couldn't cope with the thought of going to prison so he killed Nathan, killed himself and if I don't find a donor, he may yet kill me. All the stress he's put our family through also led to my mum's heart attack.

"He had no reason to do what he did, he just wanted to upset me in the worst possible way.

"He said he loved me to pieces but then he did this.

"Since the day he attacked me, it's just been one thing after another."

Tania, who is currently too sick to work at Churchill China, added: "I haven't properly grieved for Nathan because of the cancer.

"I'm up at hospital every week and fighting this disease just leaves me so tired. I can't sleep anymore. If I'm not thinking about the cancer, my thoughts turn to Nathan.

"I'm not coping very well. It's been a rollercoaster couple of years.

"I've then got all my money worries. I just don't know what more I can cope with.

"I was planning on having a 50th birthday in the new year but we've put the plans on hold because I don't know when I'll be in hospital. I wanted to something to say thank-you to all my friends and family but everything is still up in the air.

"I can't plan anything. My life has been on hold since the day that man attacked me."

Tania has monthly appointments at Christies in Manchester to check whether there is a match.

Urging people to join the register, she added: "It's just a simple test you do in your own home send off.

"I'm not doing this just for me but for everyone else who needs a donor. You never know, you could save someone's life. It's my last chance to see another Christmas."

Who can join?

Anyone aged 16 to 30 and in good health can join.

Younger donors provide better outcomes for patients, so our recruitment age range means we focus on recruiting the best possible donors.

How to join?

Fill in the online application form and Antony Nolan will send a swab pack in the post to complete and send back which will then be added to the stem cell register.

How long will I be on the register?

Those who join will remain on the register until the age of 61. If you ever come up as a match for a patient, Antony Nolan be in touch straightaway.

What happens if I'm a match?

Antony Nolan will organise everything including travel and accommodation.

How do you make a donation?

There are two ways you might be asked to donate:90% of people donate via their bloodstreamand10% have their stem cells collected via their bone marrowwhile under general anaesthetic.

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Cancer-hit mum's plea for donor after her ex murdered son who could have been match - Mirror Online

Bone Marrow Stem Cells Comments Off on Cancer-hit mum’s plea for donor after her ex murdered son who could have been match – Mirror Online | December 29th, 2019

This undated screen grab shows the cell-division of two fertilized human embryos during the first 24 hours of embryonic development following IVF treatment at a private clinic in London. ( Jim Dyson/Getty Images )

LOS ANGELES - Some of the scientific advancements of the 2010s have been truly mind-blowing, and perhaps none more so than the leaps and bounds weve made in the realm of reproduction.

This was not only the decade in which the first three-parent baby was born, it was the era when a rogue scientist chose to make edits to a set of twin girls DNA, making real the long-imagined scenario of genetically altering human beings while simultaneously thrusting the deeply complicated ethical discussions surrounding this practice into the limelight.

These are the five most life-altering breakthroughs in reproduction from the past decade.

In 2018, Chinese biophysics researcher He Jiankui announced that he had used the gene-editing tool CRISPR to modify the genes of two twin girls before birth. He and his team said that their goal was to make the girls immune to infection by HIV through the elimination of a gene called CCR5.

When the news broke, many mainstream scientists criticized the attempt, calling it too unsafe to try. Where some people saw the potential for a new kind of medical treatment capable of eradicating genetic disease, others saw a window into a dystopian future filled with designer babies and framed by a new kind of eugenics.

At the time, Dr. Kiran Musunuru, a University of Pennsylvania gene-editing expert, said Hes work was unconscionable... an experiment on human beings that is not morally or ethically defensible.

Other experts believe Hes work could propel the field of gene editing forward.

The twins, known as Lulu and Nana, have continued to make headlines since their birth. The gene modification that He claims to have carried out may have caused some unintended mutations in other parts of the genome, which could have unpredictable consequences for their health long term something many scientists who argue against Hes work cite as a reason to hold off on using gene-editing technology on humans.

Only time will tell what will happen to Lulu and Nana and if the edits to their DNA ultimately help or hurt them, but their story pushed the topic of human gene-editing and the ethics surrounding it to the forefront of the global scientific community.

In 2016, a technique called mitochondrial transfer was used successfully for the first time to create a three-parent baby grown from a fathers sperm, a mothers cell nucleus and a third donors egg that had the nucleus removed.

This technique was developed to prevent the transmission of certain genetic disorders through the mothers mitochondria. The majority of a three-parent babys DNA would come from his parents in the form of nuclear DNA, and only a small portion would come from the donor in the form of mitochondrial DNA.

A team led by physician John Zhang at the New Hope Fertility Center in New York City facilitated the birth of the first three-parent baby in April 2016.

Using human pluripotent stem cells, researchers were able to make the precursors of human sperm or eggs. In other words, they reprogrammed skin and blood stem cells to become an early-state version of what would eventually become either sperm or an egg.

"The creation of primordial germ cells is one of the earliest events during early mammalian development," Dr. Naoko Irie, first author of the paper from the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge told Science Daily. "It's a stage we've managed to recreate using stem cells from mice and rats, but until now few researches have done this systematically using human stem cells. It has highlighted important differences between embryo development in humans and rodents that may mean findings in mice and rats may not be directly extrapolated to humans."

A 2018 study showed that gene editing can allow two same-sex mice to conceive pups, and two female mice were able to successfully create healthy pups that then went on to reproduce themselves.

A team of researchers at the Chinese Academy of Sciences in Beijing, led by developmental biologist Qi Zhou, were able to use gene editing to produce 29 living mice from two females, seven of which went on to have their own pups. They were able to produce 12 pups from two male parents, but those offspring were not able to live more than two days.Whether or not the method can one day be used in same-sex human reproduction is still up for debate.

For the first time ever, Chinese scientists were able to clone two primates using the technique that produced Dolly the sheep, the first mammal to be cloned from an adult somatic cell via nuclear transfer.

The two cloned female macaques were named Zhong Zhong and Hua Hua, and their successful birth opened up the possibility of using the same cloning method to one day clone humans.

Read the rest here:
What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it - FOX 10 News Phoenix

Skin Stem Cells Comments Off on What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it – FOX 10 News Phoenix | December 29th, 2019

Fred Hutchinson Cancer Research Center researchers continued to explore the edge of human knowledge as they seek cures for cancer, HIV and other diseases. Here we highlight a sampling of the most interesting and important research from the past year.

Why does a common virus plague bone marrow transplant patients? New study challenges dogma, opens door to new therapies.

You may not have heard of cytomegalovirus, but the two of you have likely met.

In fact, odds are its dozing inside you right now.

Cytomegalovirus, or CMV, infects at least half of all adults worldwide. Most are unaware theyre infected because their healthy immune system keeps it in check. The virus slips into dormancy, becoming a passive and lifelong passenger.

But CMV can roar back to life in anyone with a compromised immune system. The results can be life-threatening, and the virus has plagued bone marrow transplant patients for decades.

Astudy in Sciencemay rewrite the story of why the virus wreaks such havoc and hint at how to stop it.

The research challenges long-held theories about how the body controls CMV. The twist: The immune systems defense against CMV isnt a solo performance. After years of studying a mouse model, a team of researchers led byDr. Geoffrey Hillshows that an unsung actor antibodies plays a vital role.

Antibodies are one of the bodys chief ways of defending itself against infection. These Y-shaped proteins can bind, like a lock and key, to bad actors and neutralize them.

Hills insight could pave the way for cheaper, safer therapies using antibodies to protect transplant patients against CMV. In a tantalizing hint, the researchers found that a dose of the right antibodies after transplantation can keep the virus dormant in mice, without the need for any other immune cells.

This is a big deal for the transplant field, said Hill, the studys senior author and director of Hematopoietic Stem Cell Transplantation at Fred Hutch. Were turning dogma on its head, and that could meet the urgent need for inexpensive and nontoxic therapies to improve patient outcomes.

Microbiome triggers top killer after bone marrow transplant and theres a potential way to stop it

Bone marrow transplants have beencuring some blood cancers for decades. But for just as long, a potentially fatal complication has lurked in the background.

In a bone marrow transplant, a patients diseased blood-forming stem cells are wiped out and then replaced by a donors healthy cells. Those donor cells are the key to the cure; they recognize and attack the patients cancer cells.

But sometimes they attack the patients healthy cells, too. This condition, called graft-vs.-host disease, can develop throughout the patients body in organs like the skin, liver, eyes and lungs.

If GVHD occurs in the gut, it can be lethal. Buthowthe disease occurs has been a mystery.

Until now.A studypublished in the journal Immunity identifies the complex chain of events that triggers GVHD in the gut. It involves a large cast of cells and molecules, including some from a surprising source: the trillions of tiny organisms that live in and on us known as the microbiome.

The scientists, led by Drs. Motoko Koyama andGeoffrey Hillof Fred Hutch, also found a promising clue as they traced the diseases complicated pathway. One of the key players in that pathway is a chemical signal called interleukin-12. By snuffing out that signal, the researchers could prevent the disease from happening in mice. They are now applying for funding to test this approach in transplant patients via a clinical trial.

For Hill and Koyama, the study caps years of experiments trying to solve this whodunit. The question was never just academic. Both have seen transplant patients suffer and die from GVHD.

Whether you live or die after a [donor] bone marrow transplant can, to a large extent, depend on whether or not you get graft-vs.-host disease of the gut, Hill said. Now that we understand that the gut both initiates and is itself the target of GVHD, we might be able to intervene to stop the whole process from starting.

High-tech approach solves real mystery in many cancers

Genetic mutations are the spark and fuel for cancer. Hundreds of DNA mutations have been linked to human cancers, and theyre easier than ever to find and catalog, thanks to new genomic technologies.

But its remained difficult to find out what those mutations are doing to drive cancer growth so that scientists can design new treatments to intervene.

In research published in the journal Nature, a coast-to-coast group of collaborators applied a powerful new method to do just that. The team showed how one commonly mutated gene actually drives cancer growth and how, potentially, to counteract it.

Even for very well-studied mutations, its frequently not obvious what the specific underlying processes are that promote cancer growth, said the studys co-leader, Dr. Robert Bradley of Fred Hutch. When we understand how to map a mutation to the development of cancer, then we can start to think about how to block that process for therapy.

The gene Bradley and collaborators studied, called SF3B1, was mutated in 19 different ways in the several different cancer types they looked at. That gene is so critical to a fundamental cell process that when it is mutated, things get screwed up all over the cell.

The biggest surprise to the scientists was that, out of all this complexity, an elegantly simple answer emerged. No matter how SF3B1 was mutated, no matter in what type of cancer they examined, no matter what else was out of whack in the cells, just one key process was central in driving cancer growth.

Once they knew what the problematic mechanism was, the scientists could intervene. In mice, implanted human tumors started to shrink when injected with the researchers custom-designed molecular repair kit.

The experimental treatment they designed is years away from human patients. For now, they hope their work prompts other researchers to study this mechanism to prove that its happening in many cancers with SF3B1 mutations.

Study reveals how blood vessels in the bone marrow protect dormant tumor cells, suggests a way to kill them in their sleep

Researchers at Fred Hutch may have found a way to essentially smother cancer cells in their sleep, preventing them from ever waking up and forming deadly metastatic tumors.

The work, led by translational researcher Dr. Cyrus Ghajar, has also turned on its ear the longstanding belief that chemotherapy cant kill dormant disseminated tumor cells cancer cells that escape early on and hide out in other regions of the body because those cells are in a sleeper state. Theyve stopped growing, so chemo which blindly targets all fast-growing cells, healthy and otherwise doesnt work.

Thats not quite the case.

Its always been assumed that dormant cells cannot be killed by any kind of chemotherapy because theyre not dividing, said Ghajar, who runs the Laboratory for the Study of Metastatic Microenvironments at Fred Hutch. But what were showing is thats not true. Theyre relying on survival signaling in their microenvironment, in this case specifically from blood vessels within the bone marrow. And if you can take away that signaling, you can sensitize them to chemotherapy.

Ghajars paper, published in Nature Cell Biology, is the culmination of more than four years work and proposes both a paradigm shift in how we view dormant disseminated tumor cells and a new therapy to potentially slay this sleeping giant. Although its still early days, Ghajar and his team slashed the metastatic relapse rate in his mice by more than two-thirds.

Cancer doesnt just spread because a primary tumor has reached a certain size or stage. Disseminated tumor cells, or DTCs, can break off before a tumor has even formed and travel to distant sites in the body where they lie dormant until something wakes them up and they start the deadly process of metastasis, or cancer spread/colonization.

One common hideout for these sleepy creeps is the bone marrow. Dormant tumor cells have been found in the bone marrow of breast cancer patients at the very earliest stage of the disease DCIS or stage 0 and Ghajar said theyre mostly likely present in other patients with early-stage disease, as well.

Past research has shown an association between DTCs in the bone marrow of cancer patients and metastatic recurrence and not necessarily just bone metastasis.

Additional recommended articles:

Failed Alzheimers drug boosts CAR T-cell therapy

Engineered immune cells get a helping hand in new clinical trial for multiple myeloma patients

They may not have made a dent against Alzheimers. But it turns out experimental drugs called gamma secretase inhibitors, or GSIs, sure can bedevil cancer. Fred Hutch research describes how GSIs can reverse a crafty disappearing act that multiple myeloma pulls on the immune system. That ability to vanish even tricks T cells that are genetically programmed to home in on and attack myeloma cells.

* * *

How to boost cancer clinical trial participation

New study suggests loosening strict comorbidity criteria would open trials to thousands of previously exempt patients

A new study led by Dr. Joseph Unger offers a tantalizing solution to low clinical trial participation: loosen up the strict eligibility criteria. Low participation is a problem thats plagued cancer researchers for decades, with most estimates putting adult cancer patient involvement at less than 5%. In many cases, the patients clinical status that is, their various medical conditions exclude them from even being considered for a trial.

* * *

Baiting for B cells: A clever new way to make an AIDS vaccine

Researchers fish for rare blood cells that can evolve into HIV blockers

Scientists at Fred Hutch have developed a new strategy to counter the frustrating ability of HIV to sidestep vaccines designed to block it. It is a scheme that relies on one of the oldest tricks in the book for a fisherman: Use the right bait. The vaccine researchers were able to use a tiny chunk of protein as bait to fish for extremely rare white blood cells hidden within ordinary blood.

* * *

Special delivery: Gold nanoparticles ship CRISPR cargo

Scientists used their new golden courier to edit genes tied to HIV, genetic blood disorders

Tiny golden delivery trucks created at Fred Hutch can ship CRISPR into human blood stem cells, offering a potential way to treat diseases like HIV and sickle cell anemia. And the researchers behind those trucks have even bigger distribution dreams.

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Immunotherapy prevents relapse in small leukemia trial

Engineered T cells kept leukemia from returning in 12 high-risk patients

The statistics are grim: For patients with high-risk acute myeloid leukemia, more than 60% will relapse within two years of a bone marrow transplant. The return of their cancer is the leading cause of death for these patients.

Butresults from a small trialof genetically modified immune cells hint at a way of protecting these patients. Scientists used engineered T cells to prevent relapse in 12 AML patients after a bone marrow transplant put their disease in remission. They all remain cancer-free after a median follow-up of more than three years.

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Nanotech turns pro-tumor immune cells into cancer-killing triple agents

Strategy doubles survival in mice with cancer

Our immune cells usually do a great job of keeping us healthy, staving off infection and killing tumor cells. But sometimes, they betray us and join the enemy: cancer. Tumors often release factors that convince immune cells to help tumors instead of hurting them. But what if these double agent immune cells could be convinced to switch allegiance yet again? Nanotechnology could be the key to redirecting specialized immune cells to attack and shrink tumors. Research showed in mice that minuscule, dissolving polymer particles can ferry genetic instructions that temporarily rewire certain immune-suppressing cells into cancer fighters without causing bodywide toxicities.

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Public health throws shade on tanning, and it works

New study shows sharp drop in melanoma rates in people under 30, but skin cancer rates still going up in those over 40

In a big win for cancer prevention, Fred Hutch and University of Washington researchers found a sustained, statistically and clinically significant downtrend in melanoma rates in people under 30 a near 25% drop over 10 years time.

Fred Hutch News Service writers Susan Keown, Daine Mapes, Jake Siegel, Sabrina Richards and Sabin Russell contributed reporting for these articles.

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Fred Hutch science and research highlights 2019 - Fred Hutch News Service

Skin Stem Cells Comments Off on Fred Hutch science and research highlights 2019 – Fred Hutch News Service | December 29th, 2019

Have you ever wondered why the first cold day of fall feels so much colder than a day the exact same temperature at the end of the winter? Its not just a matter of perspective: Your body really is more prepared for cold conditions at certain times of the yearthanks to a mysterious form of fat.

When you experience cold, your body responds in a few noticeable ways. Your blood vessels constricta process called vasoconstrictiontaking blood away from your extremities and keeping it near your core. And you shiver, meaning certain muscles start shaking to produce heat. But shivering also triggers the release of a hormone called irisin, which jump-starts a lesser-known cold weather response: the activation and buildup of brown fat.

Brown adipose tissue, or brown fat, is different from the white fat we might think of when discussing diet or weight loss. White fat lines our skin and muscles, cushioning our organs and bones. But brown fat appears only in specific areas around the neck, spine, aorta, and kidneys. It builds up in clumps around major blood vessels, warming the blood as it passes through the body. If vasoconstriction is closing the window, brown fat is turning on the heater, says Yossi Rathner, a physiologist at the University of Melbourne.

We dont create enough brown fat to cause noticeable weight gain, but the small deposits are still powerful thanks to their high concentration of energy-creating mitochondria. Instead of burning calories to produce energy to power the body, the mitochondria in brown fat burn calories to produce heat. By acting like little heating stations for blood vessels, these soft clumps of insulation help us deal with the cold more efficiently than shivering, which expends a lot of energy, and vasoconstriction, which puts us at risk of frostbite.

Your body goes from a rickety radiator to a smooth central heating system by the end of the winter, says Francesco S. Celi, a professor of medicine at Virginia Commonwealth University.

But the effect only lasts for as long as we need it. When the temperature warms up, brown fat fades away. If we are not exposed to the cold, the brown fat will atrophy, says Barbara Cannon, a physiologist at Stockholm University. There may be a few stem cells left in the area for later regeneration, but it will nearly disappear, Cannon says.

Even a one-month tropical vacation is enough to deplete ones brown fat reserves, Celi says, creating that extra cold sensation a traveler might feel upon returning to a cold climate.

You could say brown fat is winter's undercover hero, dropping in when we need it most, and disappearing once the job is done. So, the next time a chilly day leaves you shivering, just remember help is on the way.

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Brown fat warms your body in cold weather | NOVA - NOVA Next

Skin Stem Cells Comments Off on Brown fat warms your body in cold weather | NOVA – NOVA Next | December 29th, 2019