AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn2B/- spinal muscular atrophy mice

Brown, SJ; Soltic, D; Synowsky, SA; Shirran, SL; Chilcott, E; Shorrock, HK; Gillingwater, TH; Yáñez-Muñoz, RJ; Schneider, B; Bowerman, M; Fuller, HR

Fuller, HR (通讯作者)，RJAH Orthopaed Hosp, Wolfson Ctr Inherited Neuromuscular Dis, TORCH Bldg, Oswestry SY10 7AG, England.

HUMAN MOLECULAR GENETICS, 2023; 32 (20): 2950

Abstract

Structural, functional and molecular cardiac defects have been reported in spinal muscular atrophy (SMA) patients and mouse models. Previous quantitat......

Full Text Link


Links

期刊讨论 | 中国SCI论文 | 期刊主页 | 投稿经验 | 杂志官网 | 投稿链接 | 作者需知 | PMC链接 | Pubmed全文检索