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Article

Anatomically defined neuron-based rescue of neurodegenerative Niemann-Pick type C disorder.

The Journal of neuroscience : the official journal of the Society for Neuroscience - 23 Mar 2011

Lopez Manuel E, Klein Andres D, Dimbil Ubah J, Scott Matthew P

Abstract excerpt

Niemann-Pick type C disease is a fatal lysosomal storage disorder caused by loss of NPC1 function. The disorder severely affects multiple body systems, particularly the nervous system. To test whether rescue of NPC1 activity in neurons, astrocytes, or other cell types can correct the neurological defects, a Tet-inducible Npc1-YFP transgene was introduced into Npc1(-/-) mice for the cell type-specific rescue of...

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Identifiers

PMID: 21430138
DOI: 10.1523/JNEUROSCI.5981-10.2011
PMCID: PMC3071647

Genes

NPC1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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