News & Media


November 16, 2018

Big Gains in FSHD Research: A Newly Published Model of FSHD and a Potential Gene Therapy to Improve Functional Outcomes

The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.


July 24, 2018

Preclinical Gene Therapy Study Shows Muscle Restoration in Charcot-Marie-Tooth Disease Model

Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.


April 2, 2018

Can Gene Therapy Treat Dominantly Inherited Disorders?

Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.

News & Media

September 20, 2016

Nationwide Children’s Hospital Leads Effort for Gene Therapy Treatment for Duchenne Muscular Dystrophy

Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy...

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