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Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene. It is the most common, severe childhood form of muscular dystrophy. We investigated an alternative to dystrophin replacement by overexpressing ITGA7 using adeno-associated virus (AAV) delivery. ITGA7 is a laminin receptor in s...

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Dettagli Bibliografici
Pubblicato in:Hum Gene Ther
Autori principali: Heller, Kristin N., Montgomery, Chrystal L., Shontz, Kimberly M., Clark, K. Reed, Mendell, Jerry R., Rodino-Klapac, Louise R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4630797/
https://ncbi.nlm.nih.gov/pubmed/26076707
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.062
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