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Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors

Duchenne muscular dystrophy (DMD) is the most common lethal muscle disorder in children. It is caused by mutations of the dystrophin gene. Adeno-associated virus (AAV)-mediated gene replacement therapy has been actively pursued to treat DMD. However, this promising therapeutic modality has been chal...

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Detalhes bibliográficos
Main Authors: Lostal, William, Kodippili, Kasun, Yue, Yongping, Duan, Dongsheng
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2014
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4064726/
https://ncbi.nlm.nih.gov/pubmed/24580018
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.210
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