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Article

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

26 Jun 2006

Abstract excerpt

Tay-Sachs disease is a prototypic neurodegenerative disease. Lysosomal storage of GM2 ganglioside in Tay-Sachs and the related disorder, Sandhoff disease, is caused by deficiency of beta-hexosaminidase A, a heterodimeric protein. Tay-Sachs-related diseases (GM2 gangliosidoses) are incurable, but gene therapy has the potential for widespread correction of the underlying lysosomal defect by means of the...

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PMID: 16801539
DOI: 10.1073/pnas.0603765103

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