News & Media

News
November 22, 2021
Nationwide Children’s Spinoff Andelyn Biosciences to Open New Lab in Dublin
Blog
April 15, 2020
Gene therapy is the process of replacing damaged or unhealthy genetic material with new material for treating various diseases and...
Research
May 28, 2019
Researchers identify a key regulatory protein implicated in Galgt2 overexpression and begin to elucidate its protective mechanism against muscular dystrophy.
Research
April 19, 2019
Researchers recently published the long-term outcomes of patients who received the investigational drug AVXS-101 – an adeno-associated virus serotype 9 mediated gene replacement therapy.
Research
November 16, 2018
The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.
Research
July 24, 2018
Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.
Research
April 2, 2018
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
September 20, 2016
Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy...