Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial
评估 rAAVrh74.MHCK7.micro-dystrophin 全身递送治疗杜氏肌营养不良症患儿的效果:一项非随机对照试验
期刊:Jama Neurology
影响因子:21.3
doi:10.1001/jamaneurol.2020.1484
Mendell, Jerry R; Sahenk, Zarife; Lehman, Kelly; Nease, Carrie; Lowes, Linda P; Miller, Natalie F; Iammarino, Megan A; Alfano, Lindsay N; Nicholl, Amanda; Al-Zaidy, Samiah; Lewis, Sarah; Church, Kathleen; Shell, Richard; Cripe, Linda H; Potter, Rachael A; Griffin, Danielle A; Pozsgai, Eric; Dugar, Ashish; Hogan, Mark; Rodino-Klapac, Louise R