A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.
肌营养性 AAV 载体与骨骼肌顺式调控元件相结合，可改善庞贝氏症小鼠模型中的糖原清除
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101464
Sellier P, Collaud F, Benchekroun Y Krimi, Jimenez V, Leon X, Daniele N, Pham Q H, El Andari J, VandenDriessche T, Chuah M K, Grimm D, Bosch F, Mingozzi F, Ronzitti G
