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AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa

Retinitis pigmentosa (RP) consists of a group of inherited, retinal degenerative disorders and is characterized by progressive loss of rod photoreceptors and eventual degeneration of cones in advanced stages, resulting in vision loss or blindness. Gene therapy has been effective in treating autosoma...

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Detalles Bibliográficos
Autores principales: Shahin, Saba, Xu, Hui, Lu, Bin, Mercado, Augustus, Jones, Melissa K., Bakondi, Benjamin, Wang, Shaomei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9026811/
https://www.ncbi.nlm.nih.gov/pubmed/35456659
http://dx.doi.org/10.3390/pharmaceutics14040824