Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
新生儿全身基因疗法可恢复庞贝病大鼠模型的心肺功能。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.06.022
Fuller David D, Rana Sabhya, Thakre Prajwal P, Benevides Ethan S, Pope Megan K, Todd Adrian G, Jensen Victoria N, Vaught Lauren, Cloutier Denise A, Ribas Roberto A, Larson Reece C, Gentry Matthew S, Sun Ramon C, Chandran Vijay, Corti Manuela, Falk Darin J, Byrne Barry J