AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system
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Sandhoff disease (SD) is an autosomal recessive neurodegenerative disease caused by a mutation in the gene for the β-subunit of β-N-acetylhexosaminidase (Hex), resulting in the inability to catabolize ganglioside GM2 within the lysosomes. SD presents with an... ...
