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Autor:
Kathleen Church, Samiah Al-Zaidy, Ashish Dugar, Kelly Lehman, Zarife Sahenk, Rachael A Potter, Sarah Lewis, Mark J. Hogan, Louise R. Rodino-Klapac, N. Miller, Carrie Nease, Richard Shell, Amanda Nicholl, Danielle A. Griffin, Jerry R. Mendell, M. Iammarino, Lindsay N. Alfano, Eric Pozsgai, Linda H. Cripe, Linda Lowes
Publikováno v:
JAMA Neurol
Importance Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype rh74 (rAAVrh74) and codon-optimized human micro-dystrophin driven by a skeletal and