RNA interference gene therapy in dominant retinitis pigmentosa and cone-rod dystrophy mouse models caused by GCAP1 mutations

RNA interference (RNAi) knockdown is an efficacious therapeutic strategy for silencing genes causative for dominant retinal dystrophies. To test this, we used self-complementary (sc) AAV2/8 vector to develop an RNAi-based therapy in two dominant retinal degeneration mouse models. The allele-specific...

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Detalles Bibliográficos
Autores principales: Jiang, Li, Frederick, Jeanne M., Baehr, Wolfgang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2014
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3985072/
https://www.ncbi.nlm.nih.gov/pubmed/24778606
http://dx.doi.org/10.3389/fnmol.2014.00025

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3985072/
https://www.ncbi.nlm.nih.gov/pubmed/24778606
http://dx.doi.org/10.3389/fnmol.2014.00025

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