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

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
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Adeno-associated virus
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container_end_page e17
container_issue 9
container_start_page e17
container_title Molecular therapy
container_volume 21
creator Wicki, Jacqueline
Banks, Glen B
Ng, Rainer
Allen, James
Finn, Eric E
Nguyen, Quynh V
Hauschka, Stephen D
Odom, Guy L
Chamberlain, Jeffrey S
description 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 patients regardless of their underlying genetic mutation. We have previously shown that highly truncated dystrophins can significantly ameliorate the dystrophic phenotype in small and large animal models of DMD following delivery with recombinant AAV vectors. The data suggest that the optimized microdystrophin cassette is promising for use in human clinical trials of gene therapy for DMD.
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source Open Access: PubMed Central; Free E-Journal (出版社公開部分のみ）; Alma/SFX Local Collection; ProQuest Central
subjects Adeno-associated virus
title Development of a Clinical AAV-Based Vector Encoding a Microdystrophin Transgene for the Treatment of Duchenne Muscular Dystrophy
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