Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I
FKRP AAV 基因治疗联合核糖醇治疗 LGMD2I 疗效提高
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2023.10.022
Marcela P Cataldi, Charles H Vannoy, Anthony Blaeser, Jason D Tucker, Victoria Leroy, Raegan Rawls, Jessalyn Killilee, Molly C Holbrook, Qi Long Lu