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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Detalles Bibliográficos
Autores principales: Nasir, Gibran, Chopra, Rajiv, Elwood, Fiona, Ahmed, Seemin S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Medicine
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

Internet

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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