Tag Archives: The Stem Cell Blog

Baby gene therapy study offers hope for fatal muscle disease – Bennington Banner

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2017-11-06 NEWS

Bennington Banner

Baby gene therapy study offers hope for fatal muscle disease
Bennington Banner
Dr. Jerry Mendell of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio. Mendell led a small study of gene therapy in babies born with a usually fatal neuromuscular disease and reported Nov. 1 that the experiment extended

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2017-11-06 NEWS

Bennington Banner

Baby gene therapy study offers hope for fatal muscle disease
Bennington Banner
Dr. Jerry Mendell of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio. Mendell led a small study of gene therapy in babies born with a usually fatal neuromuscular disease and reported Nov. 1 that the experiment extended

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2017-11-06 NEWS

Sarepta Therapeutics (SRPT): FDA Clears GALGT2 Gene Therapy Program – William Blair
StreetInsider.com
William Blair analyst, Tim Lugo, reiterated his Outperform rating on shares of Sarepta Therapeutics (NASDAQ: SRPT) after the company announced the FDA had approved an investigational new drug (IND) application for its GALGT2 gene therapy program …
Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin GeneGlobeNewswire (press release)

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2017-11-06 NEWS

pharmaphorum

Novartis files CART in Europe in broader use than Gilead rival
pharmaphorum
Chimeric antigen receptor Tcell (CAR-T) therapies are one-off treatments that involve harvesting a patient’s Tcells, and genetically reprogramming them to fight cancer. Trial data so far show they offer a complete response – a complete absence of
Novartis files CART therapy in EuropePharmaTimes
Novartis seeks European approval for cell therapy KymriahReuters

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2017-11-06 NEWS

CBS News

Playing God: “We are in the midst of a genetic revolution”
CBS News
Dr. Emmanuelle Charpintier at the Max Planck Institute in Germany is one half of the duo that discovered CRISPR‘s gene-cutting abilities. She and her research partner, Jennifer Doudna of UC-Berkeley, manipulated a naturally occurring defense system in …