In vivo base editing rescues photoreceptors in a mouse model of retinitis pigmentosa

Retinitis pigmentosa (RP) is a group of retinal diseases that cause the progressive death of retinal photoreceptor cells and eventually blindness. Mutations in the β-domain of the phosphodiesterase 6 (Pde6b) gene are the most identified causes of autosomal recessive RP. Clinically, there is no effec...

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Detalles Bibliográficos
Autores principales: Su, Jing, She, Kaiqin, Song, Li, Jin, Xiu, Li, Ruiting, Zhao, Qinyu, Xiao, Jianlu, Chen, Danian, Cheng, Hui, Lu, Fang, Wei, Yuquan, Yang, Yang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9996133/
https://www.ncbi.nlm.nih.gov/pubmed/36910709
http://dx.doi.org/10.1016/j.omtn.2023.02.011

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