Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing

Mutations in the gene for Retinitis Pigmentosa GTPase Regulator (RPGR) cause the X-linked form of inherited retinal degeneration, and the majority are frameshift mutations in a highly repetitive, purine-rich region of RPGR known as the OFR15 exon. Truncation of the reading frame in this terminal exo...

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Detalles Bibliográficos
Autores principales: Gumerson, Jessica D., Alsufyani, Amal, Yu, Wenhan, Lei, Jingqi, Sun, Xun, Dong, Lijin, Wu, Zhijian, Li, Tiansen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8856954/
https://www.ncbi.nlm.nih.gov/pubmed/34257417
http://dx.doi.org/10.1038/s41434-021-00258-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8856954/
https://www.ncbi.nlm.nih.gov/pubmed/34257417
http://dx.doi.org/10.1038/s41434-021-00258-6

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