Dystrophin Immunity in Duchenne’s Muscular Dystrophy

We report on delivery of a functional dystrophin transgene to skeletal muscle in six patients with Duchenne’s muscular dystrophy. Dystrophin-specific T cells were detected after treatment, providing evidence of transgene expression even when the functional protein was not visualized in skeletal musc...

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Bibliografiske detaljer
Main Authors: Mendell, Jerry R., Campbell, Katherine, Rodino-Klapac, Louise, Sahenk, Zarife, Shilling, Chris, Lewis, Sarah, Bowles, Dawn, Gray, Steven, Li, Chengwen, Galloway, Gloria, Malik, Vinod, Coley, Brian, Clark, K. Reed, Li, Juan, Xiao, Xiao, Samulski, Jade, McPhee, Scott W., Samulski, R. Jude, Walker, Christopher M.
Format: Artigo
Sprog:Inglês
Udgivet: 2010
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3014106/
https://ncbi.nlm.nih.gov/pubmed/20925545
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1000228
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