Intrathecal delivery of AAVrh10-mHexa combined with anti-inflammatory treatment reduces neuropathological markers and extends the lifespan of mice with early-onset Tay-Sachs disease
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Tay-Sachs disease is a lysosomal storage disorder caused by mutations in the HEXA gene, which encodes the α-subunit of β-hexosaminidase A-an enzyme that breaks down GM2 ganglioside. Recently, a mouse model of Tay-Sachs, the DKO, with deficiencies in both Hex... ...
