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Article

AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.

Investigative ophthalmology & visual science - 20 Mar 2014

Dai Xufeng, Han Juanjuan, Qi Yan, Zhang Hua, Xiang Lue, Lv Jineng, Li Jie, Deng Wen-Tao, Chang Bo, Hauswirth William W, Pang Ji-jing

Abstract excerpt

PURPOSE: The retinal degeneration 11 (rd11) mouse is a newly discovered, naturally occurring animal model with early photoreceptor dysfunction and rapid rod photoreceptor degeneration followed by cone degeneration. The rd11 mice carry a spontaneous mutation in the lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene. Here, we evaluate whether gene replacement therapy using the fast-acting tyrosine-capsid...

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Identifiers

PMID: 24557352
DOI: 10.1167/iovs.13-13654
PMCID: PMC3968931

Genes

ERG
LPCAT1

Provenance

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