Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

Globoid cell leukodystrophy (GLD) or Krabbe disease is a neurodegenerative disorder caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This deficiency results in accumulation of certain galactolipids including psychosine which is cytotoxic for myelin-producing cells. Treat...

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Detalhes bibliográficos
Main Authors: Rafi, Mohammad A, Rao, Han Zhi, Luzi, Paola, Curtis, Mark T, Wenger, David A
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3498799/
https://ncbi.nlm.nih.gov/pubmed/22850681
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.153
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