Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia

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...

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Detalles Bibliográficos
Publicado en:PLoS One
Autores principales: Dai, Xufeng, He, Ying, Zhang, Hua, Zhang, Yangyang, Liu, Yan, Wang, Muran, Chen, Hao, Pang, Ji-jing
Formato: Artigo
Lenguaje:Inglês
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5683625/
https://ncbi.nlm.nih.gov/pubmed/29131863
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0188032
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