Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.
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Childers MK, et al. Among authors: guan x.
Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523.
Sci Transl Med. 2014.
PMID: 24452262
Free PMC article.