Search Results for: stem cells for ms

Mesoblast gets FDA RMAT; List of 10 total designations so far

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Mesoblast-e1514313188176

Australian stem cell biotech Mesoblast announced that it has received regenerative medicine advanced therapy (RMAT) designation from the U.S. FDA. This is very good news for the company and an encouraging development for the field. Interestingly, last month the FDA clarified that there is expanded RMAT designation that can include gene therapies too. At the Meeting […]

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Seeing isn’t always believing: a cautionary tale on GFP transfer when trying to restore vision

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Valerie-Wallace

By Samantha Yammine When our TV stops working, it’s usually because one of the wires has come unplugged. We begrudgingly huff and puff over to the back of the TV, track down the loose culprit, plug it back in, and boom: back to Netflix. If you multiply all those wires in the back of your

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On the threshold of cell therapy for Parkinson’s disease

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Dopamine-neurons-derived-from-a-Parkinsons-disease-patients-iPSCs-Tyrosine-hydroxylase-1

By Jeanne Loring There are ten million people in the world who have Parkinson’s disease. 125,000 of these are living in California.  People with the disease often have to step away from their jobs because the main symptoms – tremor or freezing up of muscles – make it difficult to get through a whole day

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7 tech hurdles to human germline CRISPR

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Human-crispr-challenges

Human germline CRISPR raises major bioethical considerations, but what about technical issues? Setting aside the many ethical issue about the general idea of human modification itself, could this really work? Yes in theory it could, but there are some very tough technological challenges that could and likely would cause failures or unacceptable outcomes at many steps

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Cord blood for cerebral palsy: mostly discouraging new trial data

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umbilical-cord-blood, umbilical cord blood stem cells

Some notable newly published Phase II data from the Duke cord blood for cerebral palsy trial unfortunately doesn’t give much reason for optimism that this approach is going to have a substantial positive effect for these kids. The double-blinded, placebo-controlled study did not find a meaningful benefit overall from infusions of autologous cord blood (ACB) for

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Countering that Pro-Heritable Human CRISPR WSJ Piece

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human-embryo-modification

It’s germline, heritable human CRISPR time, right? Wrong. But the particularly enthusiastic supporters of heritable human CRISPR often cite hypothetical benefits in glowing terms, but either don’t mention risks or strongly downplay them. These fans also tend to leave alternative, proven and safe technologies such as preimplantation genetic diagnosis (PGD) out of the discussion or

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1st Knockout Human Embryos Made with CRISPR: My Take on the Pub

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Fogarty-Fig-3d-human-embryo-CRISPR-e1505774206656

Scientist make knockout human embryos with CRISPR? Today we see a new Nature paper (Fogarty, et al.) on CRISPR “gene editing” of human embryos, this time from the UK from Kathy Niakan’s group. Niakan got UK permission about 18 months ago to CRISPR healthy human embryos so they’ve been hard at work since. Because Fredrik

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Young blood as anti-aging fountain of youth: hype or hope?

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There’s an idea floating around out there called “young blood” to use donations from young folks to infuse older people to reverse aging. I’m skeptical. Imagine an old man and a teenager sitting side by side, with blood flowing from the kid to the oldster in a stab at anti-aging. Sort of like a one-way

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Reading the tea leaves as new FDA commish Gottlieb blogs on regenerative medicine

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Dr.-Scott-Gottlieb

New FDA Commissioner Dr. Scott Gottlieb, M.D., has in the past touched on stem cells and regenerative medicine therapies in speeches or written comments prior to starting his tenure at the agency. Now that he is Commissioner, he is poised to have direct impact on our field rather quickly and potentially with major changes in

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