Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
Krabbe Disease (KD) is an autosomal metabolic disorder that affects both the central and peripheral nervous systems. It is caused by a functional deficiency of the lysosomal enzyme, galactocerebrosidase (GALC), resulting in an accumulation of the toxic metabolite, psychosine. Psychosine accumulation...
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| Auteurs principaux: | , , , |
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| Format: | article |
| Langue: | EN |
| Publié: |
Frontiers Media S.A.
2021
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| Accès en ligne: | https://doaj.org/article/075dde463a2647d8bec66eeb56a8de50 |
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