AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)

Globoid cell leukodystrophy (GLD), or Krabbe disease, is an inherited, neurologic disorder that results from deficiency of a lysosomal enzyme, galactosylceramidase. Most commonly, deficits of galactosylceramidase result in widespread central and peripheral nervous system demyelination and death in a...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Bradbury, Allison M., Rafi, Mohammed A., Bagel, Jessica H., Brisson, Becky K., Marshall, Michael S., Pesayco Salvador, Jill, Jiang, Xuntain, Swain, Gary P., Prociuk, Maria L., ODonnell, Patricia A., Fitzgerald, Caitlin, Ory, Daniel S., Bongarzone, Ernesto R., Shelton, G. Diane, Wenger, David A., Vite, Charles H.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2018
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6066194/
https://ncbi.nlm.nih.gov/pubmed/29316812
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.151
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