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Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS, Li J, Wang B, Monahan PE, Rabinowitz JE, Grieger JC, Govindasamy L, Agbandje-McKenna M, Xiao X, Samulski RJ. Bowles DE, et al. Among authors: grieger jc. Mol Ther. 2012 Feb;20(2):443-55. doi: 10.1038/mt.2011.237. Epub 2011 Nov 8. Mol Ther. 2012. PMID: 22068425 Free PMC article. Clinical Trial.
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