Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa

Mutations in PRPF31 cause autosomal dominant retinitis pigmentosa, an untreatable form of blindness. Gene therapy is a promising treatment for PRPF31-retinitis pigmentosa, however, there are currently no suitable animal models in which to develop AAV-mediated gene augmentation. Here we establish Prp...

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Detalles Bibliográficos
Autores principales: Xi, Zhouhuan, Vats, Abhishek, Sahel, José-Alain, Chen, Yuanyuan, Byrne, Leah C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9744804/
https://www.ncbi.nlm.nih.gov/pubmed/36509783
http://dx.doi.org/10.1038/s41467-022-35361-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9744804/
https://www.ncbi.nlm.nih.gov/pubmed/36509783
http://dx.doi.org/10.1038/s41467-022-35361-8

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