Heat shock protein amplification improves cerebellar myelination in the Npc1nih mouse modelResearch in context

Summary: Background: Niemann-Pick disease type C (NPC) is a rare prematurely fatal lysosomal lipid storage disease with limited therapeutic options. The prominent neuropathological hallmarks include hypomyelination and cerebellar atrophy. We previously demonstrated the efficacy of recombinant human...

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Bibliographic Details
Main Authors: James Gray, María E. Fernández-Suárez, Maysa Falah, David Smith, Claire Smith, Ecem Kaya, Ashley M. Palmer, Cathrine K. Fog, Thomas Kirkegaard, Frances M. Platt
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:EBioMedicine
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2352396422005564