Article
Biology and potential strategies for the treatment of GM2 gangliosidoses.
Molecular medicine today - 1 Apr 1998
Chavany C, Jendoubi M
Abstract excerpt
The GM2 gangliosidoses are a group of heritable neurodegenerative disorders caused by excessive accumulation of the ganglioside GM2 owing to deficiency in beta-hexosaminidase activity. Tay-Sachs and Sandhoff diseases have similar clinical phenotypes resulting from a deficiency in human hexosaminidase alpha and beta subunits, respectively. The lack of treatment for GM2 gangliosidoses stimulated interest in...
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