AAV9-coGLB1 Improves Lysosomal Storage and Rescues Central Nervous System Inflammation in a Mutant Mouse Model of GM1 Gangliosidosis

Liu, SC; Ma, WH; Feng, YY; Zhang, Y; Jia, XF; Tang, CF; Tang, F; Wu, XB; Huang, YL

Huang, YL (通讯作者)，Guangzhou Med Univ, Guangzhou Women & Childrens Med Ctr, Dept Guangzhou Newborn Screening Ctr, Guangzhou 510623, Guangdong, Peoples R China.;Wu, XB (通讯作者)，Beijing Ruicy Gene Therapy Inst Rare Dis, Beijing, Peoples R China.

CURRENT GENE THERAPY, 2022; 22 (4): 352

Abstract

Background: GM1 gangliosidosis (GM1) is an autosomal recessive disorder characterized by the deficiency of beta-galactosidase (beta-gal), a ubiquitous......

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