AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD.
AAV介导的新型微型肌营养不良蛋白基因转移可改善DMD小鼠模型的病理并增强肌肉功能。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2026.102901
Owusu Lawrence, Kim SunJung, Patel Hiren, Foltz Steven, Chan Gary N Y, Kim Kwi Hye, Kopen Aaron, Yang Lin, Lawlor Michael W, Buss Nicholas, Cunningham Justine J, Liu Ye, Danos Olivier, Fiscella Michele