Phase 1 gene therapy for duchenne muscular dystrophy using a translational optimized AAV vector

Dawn E. Bowles, Scott W.J. McPhee, Chengwen Li, Steven J. Gray, Jade J. Samulski, Angelique S. Camp, Juan Li, Bing Wang, Paul E. Monahan, Joseph E. Rabinowitz, Joshua C. Grieger, Lakshmanan Govindasamy, Mavis Agbandje-Mckenna, Xiao Xiao, R. Jude Samulski

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