Development of a Clinical AAV-Based Vector Encoding a Microdystrophin Transgene for the Treatment of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a recessively-inherited muscle wasting disorder caused by mutations within the dystrophin gene. Gene therapy using adeno-associated viral (AAV) vectors to deliver dystrophin expression cassettes remains a promising treatment option that would be applicable to all...

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Veröffentlicht in:	Molecular therapy 2013-09, Vol.21 (9), p.e17-e17
Hauptverfasser:	Wicki, Jacqueline, Banks, Glen B, Ng, Rainer, Allen, James, Finn, Eric E, Nguyen, Quynh V, Hauschka, Stephen D, Odom, Guy L, Chamberlain, Jeffrey S
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus
Online-Zugang:	Volltext
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