Delivery of AAV2/9-Microdystrophin Genes Incorporating Helix 1 of the Coiled-Coil Motif in the C-Terminal Domain of Dystrophin Improves Muscle Pathology and Restores the Level of α1-Syntrophin and α-Dystrobrevin in Skeletal Muscles of mdx Mice

Duchenne muscular dystrophy is a severe X-linked inherited muscle wasting disorder caused by mutations in the dystrophin gene. Adeno-associated virus (AAV) vectors have been extensively used to deliver genes efficiently for dystrophin expression in skeletal muscles. To overcome limited packaging cap...

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Autors principals: Koo, Taeyoung, Malerba, Alberto, Athanasopoulos, Takis, Trollet, Capucine, Boldrin, Luisa, Ferry, Arnaud, Popplewell, Linda, Foster, Helen, Foster, Keith, Dickson, George
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2011
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Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3225045/
https://ncbi.nlm.nih.gov/pubmed/21453126
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.020
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