CRISPR/Cas9-mediated generation of a human induced pluripotent stem cell line with PRPF6 c.2699 G > A mutation to model retinitis pigmentosa.

Stem Cell Res

Aier Eye Institute, Changsha 410015, China; Aier Academy of Ophthalmology, Central South University, Changsha 410015, China; Aier Eye Hospital, Jinan University, Guangzhou 510632, China. Electronic address:

Published: December 2024

PRPF6, located on chromosome 20, is required for the formation of the spliceosome. Mutations in the PRPF6 gene can lead to retinitis pigmentosa (RP), a common inherited retinal disease characterized by progressive degeneration of retinal pigment epithelium and photoreceptors. Here, we generated an induced pluripotent stem cell (iPSC) line carrying the PRPF6 c.2699 G > A mutation using CRISPR/Cas9 technology, which will provide a valuable resource for RP pathogenesis and treatment research.

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http://dx.doi.org/10.1016/j.scr.2024.103581DOI Listing

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