Genetics and Therapies for GM2 Gangliosidosis

Cachon-Gonzalez, MB; Zaccariotto, E; Cox, TM

Cachon-Gonzalez, MB (reprint author), Univ Cambridge, Dept Med, Addenbrookes Hosp, Level 5,POB 157,Hills Rd, Cambridge CB2 0QQ, England.

CURRENT GENE THERAPY, 2018; 18 (2): 68

Abstract

Tay-Sachs disease, caused by impaired beta-N-acetylhexosaminidase activity, was the first GM2 gangliosidosis to be studied and one of the most severe ......

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