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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Detaylı Bibliyografya
Yayımlandı:Hum Gene Ther
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2018
Konular:
Research Articles
Online Erişim: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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