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BioWorld - Thursday, July 9, 2026
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Home » CSF-delivered AAV-based gene therapy rescues ocular symptoms in model of Tay-Sachs disease
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Ocular

CSF-delivered AAV-based gene therapy rescues ocular symptoms in model of Tay-Sachs disease

June 23, 2026
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Deficiencies of the enzyme β-N-acetylhexosaminidase (Hex) cause rare, autosomal recessive, fatal, neurodegenerative lysosomal storage disorders called GM2 gangliosidoses, including Tay-Sachs disease (TSD) and Sandhoff disease. Hex enzyme is a heterodimer encoded by HEXA (α subunit) and HEXB (β subunit), whose mutations result in TSD and Sandhoff disease, respectively.
BioWorld Science Genetic/congenital Ocular Gene therapy

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