Gene therapy ameliorates neuromuscular pathology in CLN3 disease
基因疗法可改善CLN3疾病的神经肌肉病理。
期刊:Acta Neuropathologica Communications
影响因子:5.7
doi:10.1186/s40478-025-02059-z
Ziółkowska, Ewa A; Jablonka-Shariff, Albina; Williams, Letitia L; Jansen, Matthew J; Wang, Sophie H; Eultgen, Elizabeth M; Wood, Matthew D; Hunter, Daniel A; Sharma, Jaiprakash; Sardiello, Marco; Reese, Robyn; Pestronk, Alan; Sands, Mark S; Snyder-Warwick, Alison K; Cooper, Jonathan D