AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis.
Buj-Bello A, Fougerousse F, Schwab Y, Messaddeq N, Spehner D, Pierson CR, Durand M, Kretz C, Danos O, Douar AM, Beggs AH, Schultz P, Montus M, Denèfle P, Mandel JL.
Hum Mol Genet. 2008 Jul 15;17(14):2132-43. Epub 2008 Apr 22.PMID: 18434328 [PubMed - indexed for MEDLINE]Related articlesFree article