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Article

Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 7 Oct 2020

Lahey Hannah G, Webber Chelsea J, Golebiowski Diane, Izzo Cassandra M, Horn Erin, Taghian Toloo, Rodriguez Paola, Batista Ana Rita, Ellis Lauren E, Hwang Misako, Martin Douglas R, Gray-Edwards Heather, Sena-Esteves Miguel

Abstract excerpt

The GM2 gangliosidoses, Tay-Sachs disease (TSD) and Sandhoff disease (SD), are fatal lysosomal storage disorders caused by mutations in the HEXA and HEXB genes, respectively. These mutations cause dysfunction of the lysosomal enzyme β-N-acetylhexosaminidase A (HexA) and accumulation of GM2 ganglioside (GM2) with ensuing neurodegeneration, and death by 5 years of age. Until recently, the most successful therapy...

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Identifiers

PMID: 32592687
DOI: 10.1016/j.ymthe.2020.06.021
PMCID: PMC7544971

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