Sustained alpha-sarcoglycan gene expression following gene transfer in LGMD2D

OBJECTIVE: The aim of this study was to attain long-lasting alpha-sarcoglycan gene expression in LGMD2D subjects mediated by adeno-associated virus (AAV) gene transfer under control of a muscle specific promoter (tMCK) METHODS: rAAV1.tMCK.hSGCA (3.25 × 10(11) vg) was delivered to the extensor digito...

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Auteurs principaux: Mendell, Jerry R., Rodino-Klapac, Louise R., Rosales, Xiomara Q., Coley, Brian D., Galloway, Gloria, Lewis, Sarah, Malik, Vinod, Shilling, Chris, Byrne, Barry J., Conlon, Thomas, Campbell, Katherine J., Bremer, William G., Taylor, Laura E., Flanigan, Kevin M., Kota, Janaiah, Sahenk, Zarife, Walker, Christopher M., Clark, K. Reed
Format: Artigo
Langue:Inglês
Publié: 2010
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC2970162/
https://ncbi.nlm.nih.gov/pubmed/21031578
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.22251
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