AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model
针对SMARD1/CMT2S的AAV9基因治疗优化：在SMARD1临床前模型中比较两种载体的安全性和长期疗效
期刊:Journal of Biomedical Science
影响因子:12.1
doi:10.1186/s12929-025-01204-z
Pagliari, Elisa; Anastasia, Alessia; Bellandi, Floriana; Garbellini, Manuela; Ongaro, Jessica; Taiana, Michela; Comi, Giacomo P; Ottoboni, Linda; Sierra-Delgado, Julieth Andrea; Likhite, Shibi; Meyer, Kathrin C; Nizzardo, Monica; Corti, Stefania P
