Article
In Niemann-Pick C1 mouse models, glial-only expression of the normal gene extends survival much further than do changes in genetic background or treatment with hydroxypropyl-beta-cyclodextrin.
Gene - 15 Feb 2018
Marshall Craig A, Watkins-Chow Dawn E, Palladino Giampiero, Deutsch Gail, Chandran Keshav, Pavan William J, Erickson Robert P
Abstract excerpt
The Npc1nmf164 allele of Npc1 provides a mouse model for Niemann-Pick disease type C1 (NPC1), a genetic disease known to have a widely variable phenotype. The transfer of the Npc1nmf164 mutation from the C57BL/6J inbred strain to the BALB/cJ inbred strain increased the mean lifespan from 117.8days to 153.1days, confirming that the severity of the NPC1 phenotype is strongly influenced by genetic background. The...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.