Genetic dissection of a cell-autonomous neurodegenerative disorder: lessons learned from mouse models of Niemann-Pick disease type C

Understanding neurodegenerative disease progression and its treatment requires the systematic characterization and manipulation of relevant cell types and molecular pathways. The neurodegenerative lysosomal storage disorder Niemann-Pick disease type C (NPC) is highly amenable to genetic approaches t...

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Bibliographic Details
Main Authors: Manuel E. Lopez, Matthew P. Scott
Format: Article
Language:English
Published: The Company of Biologists 2013-09-01
Series:Disease Models & Mechanisms
Online Access:http://dmm.biologists.org/content/6/5/1089