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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Publicat a:Hum Gene Ther
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2018
Matèries:
Research Articles
Accés en línia: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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