Article

Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

2021-12-23

Abstract excerpt

<h4>ABSTRACT</h4> The GM2 gangliosidoses Tay-Sachs disease and Sandhoff disease (SD) are respectively caused by mutations in the HEXA and HEXB genes encoding the α and β subunits of β-N-acetylhexosaminidase (Hex). The consequential accumulation of ganglioside in the brain leads to severe and progressive neurological impairment. There are currently no approved therapies to counteract or reverse the effects of GM2...

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Literature Corpus work: 2b084127-5e67-5164-a69f-7ec190c9f35e
DOI: 10.1101/2021.12.22.473777

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