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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...
Autores principales: | Nasir, Gibran, Chopra, Rajiv, Elwood, Fiona, Ahmed, Seemin S. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8633897/ https://www.ncbi.nlm.nih.gov/pubmed/34869463 http://dx.doi.org/10.3389/fmed.2021.760236 |
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