Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice.
双重 FKRP/FST 基因疗法可使 LGMDR9 小鼠恢复正常行走能力，增强力量，减少病理变化，并增强基因表达
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.06.028
Lam Patricia, Zygmunt Deborah A, Ashbrook Anna, Bennett Macey, Vetter Tatyana A, Martin Paul T
