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Genetic and pharmacological evidence implicates cathepsins in Niemann-Pick C cerebellar degeneration

Niemann-Pick C1 (NPC) disease, an autosomal recessive lipid trafficking disorder caused by loss-of-function mutations in the NPC1 gene, is characterized by progressive neurodegeneration resulting in cognitive impairment, ataxia and early death. Little is known about the cellular pathways leading to...

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Detalles Bibliográficos
Publicado en:Hum Mol Genet
Main Authors: Chung, Chan, Puthanveetil, Prasanth, Ory, Daniel S., Lieberman, Andrew P.
Formato: Artigo
Idioma:Inglês
Publicado: Oxford University Press 2016
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4787909/
https://ncbi.nlm.nih.gov/pubmed/26908626
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddw025
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