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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...

Topics

Animals
Behavior, Animal
Capsid
Color Vision Defects
Cyclic Nucleotide-Gated Cation Channels
Dependovirus
Disease Models, Animal
Electroretinography
Genetic Therapy
Genetic Vectors
Mice

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Mutation

Retina

Source

Open in PubMed Open full text

Identifiers

PMID: 29131863
DOI: 10.1371/journal.pone.0188032
PMCID: PMC5683625

Genes

CNGA3
ERG

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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