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Article

Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

24 Oct 2016

Abstract excerpt

Niemann-Pick disease, type C1 (NPC1) is a heritable lysosomal storage disease characterized by a progressive neurological degeneration that causes disability and premature death. A murine model of NPC1 disease (Npc1-/-) displays a rapidly progressing form of NPC1 disease which is characterized by weight loss, ataxia, increased cholesterol storage, loss of cerebellar Purkinje neurons and early lethality. To test...

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PMID: 27798114
DOI: 10.1093/hmg/ddw367

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