AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model
AAV5-miHTT 基因疗法在亨廷顿病小型猪模型中表现出广泛分布和强效降低人类突变型亨廷顿蛋白的作用
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2018.06.021
Melvin M Evers, Jana Miniarikova, Stefan Juhas, Astrid Vallès, Bozena Bohuslavova, Jana Juhasova, Helena Kupcova Skalnikova, Petr Vodicka, Ivona Valekova, Cynthia Brouwers, Bas Blits, Jacek Lubelski, Hana Kovarova, Zdenka Ellederova, Sander J van Deventer, Harald Petry, Jan Motlik, Pavlina Konstanti