A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases

The GM2-gangliosidoses are neurological diseases causing premature death, thus developing effective treatment protocols is urgent. GM2-gangliosidoses result from deficiency of a lysosomal enzyme β-hexosaminidase (Hex) and subsequent accumulation of GM2 gangliosides. Genetic changes in HEXA, encoding...

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Publicado no:Gene Ther
Main Authors: Ou, Li, Przybilla, Michael J, Tăbăran, Alexandru-Flaviu, Overn, Paula, O’Sullivan, M. Gerard, Jiang, Xuntian, Sidhu, Rohini, Kell, Pamela J., Ory, Daniel S., Whitley, Chester B.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7260097/
https://ncbi.nlm.nih.gov/pubmed/31896760
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-019-0120-5
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