Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

Globoid cell leukodystrophy (GLD; Krabbe disease) is a progressive, incurable neurodegenerative disease caused by deficient activity of the hydrolytic enzyme galactosylceramidase (GALC). The ensuing cytotoxic accumulation of psychosine results in diffuse central and peripheral nervous system (CNS, P...

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Detalhes bibliográficos
Publicado no:J Clin Invest
Main Authors: Bradbury, Allison M., Bagel, Jessica H., Nguyen, Duc, Lykken, Erik A., Pesayco Salvador, Jill, Jiang, Xuntian, Swain, Gary P., Assenmacher, Charles A., Hendricks, Ian J., Miyadera, Keiko, Hess, Rebecka S., Ostrager, Arielle, ODonnell, Patricia, Sands, Mark S., Ory, Daniel S., Shelton, G. Diane, Bongarzone, Ernesto R., Gray, Steven J., Vite, Charles H.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2020
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7456224/
https://ncbi.nlm.nih.gov/pubmed/32773406
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI133953
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