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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Autores principales:	Gibran Nasir, Rajiv Chopra, Fiona Elwood, Seemin S. Ahmed
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	Krabbe disease (globoid cell leukodystrophy) leukodystrophies adeno-associated virus gene therapy galactocerebrosidase psychosine Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/075dde463a2647d8bec66eeb56a8de50
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spelling	oai:doaj.org-article:075dde463a2647d8bec66eeb56a8de502021-11-12T13:24:16ZKrabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy2296-858X10.3389/fmed.2021.760236https://doaj.org/article/075dde463a2647d8bec66eeb56a8de502021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fmed.2021.760236/fullhttps://doaj.org/toc/2296-858XKrabbe 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 affects many different cellular pathways, leading to severe demyelination. Although there is currently no effective therapy for Krabbe disease, recent gene therapy-based approaches in animal models have indicated a promising outlook for clinical treatment. This review highlights recent findings in the pathogenesis of Krabbe disease, and evaluates AAV-based gene therapy as a promising strategy for treating this devastating pediatric disease.Gibran NasirRajiv ChopraFiona ElwoodSeemin S. AhmedFrontiers Media S.A.articleKrabbe disease (globoid cell leukodystrophy)leukodystrophiesadeno-associated virusgene therapygalactocerebrosidasepsychosineMedicine (General)R5-920ENFrontiers in Medicine, Vol 8 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Krabbe disease (globoid cell leukodystrophy) leukodystrophies adeno-associated virus gene therapy galactocerebrosidase psychosine Medicine (General) R5-920
spellingShingle	Krabbe disease (globoid cell leukodystrophy) leukodystrophies adeno-associated virus gene therapy galactocerebrosidase psychosine Medicine (General) R5-920 Gibran Nasir Rajiv Chopra Fiona Elwood Seemin S. Ahmed Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
description	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 affects many different cellular pathways, leading to severe demyelination. Although there is currently no effective therapy for Krabbe disease, recent gene therapy-based approaches in animal models have indicated a promising outlook for clinical treatment. This review highlights recent findings in the pathogenesis of Krabbe disease, and evaluates AAV-based gene therapy as a promising strategy for treating this devastating pediatric disease.
format	article
author	Gibran Nasir Rajiv Chopra Fiona Elwood Seemin S. Ahmed
author_facet	Gibran Nasir Rajiv Chopra Fiona Elwood Seemin S. Ahmed
author_sort	Gibran Nasir
title	Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
title_short	Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
title_full	Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
title_fullStr	Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
title_full_unstemmed	Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
title_sort	krabbe disease: prospects of finding a cure using aav gene therapy
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/075dde463a2647d8bec66eeb56a8de50
work_keys_str_mv	AT gibrannasir krabbediseaseprospectsoffindingacureusingaavgenetherapy AT rajivchopra krabbediseaseprospectsoffindingacureusingaavgenetherapy AT fionaelwood krabbediseaseprospectsoffindingacureusingaavgenetherapy AT seeminsahmed krabbediseaseprospectsoffindingacureusingaavgenetherapy
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Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy

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