Article
Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia.
PloS one - 1 Jan 2017
Dai Xufeng, He Ying, Zhang Hua, Zhang Yangyang, Liu Yan, Wang Muran, Chen Hao, Pang Ji-Jing
Abstract excerpt
Adeno-associated virus (AAV) vectors are important gene delivery tools for the treatment of many recessively inherited retinal diseases. For example, a wild-type (WT) AAV5 vector can deliver a full-length Cnga3 (cyclic nucleotide-gated channel alpha-3) cDNA to target cells of the cone photoreceptor function loss 5 (cpfl5) mouse, a spontaneous animal model of achromatopsia with a Cnga3 mutation. Gene therapy...
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