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

Pub. Date : 2011 Mar 23

PMID : 21430138






1 Functional Relationships(s)
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1 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 NPC1 loss. tetramethylenedisulfotetramine NPC intracellular cholesterol transporter 1 Mus musculus