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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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| Publié dans: | Gene Ther |
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| Auteurs principaux: | , , , , , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2020
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| Sujets: | |
| Accès en ligne: | 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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