Generation of a gene-corrected isogenic human iPS cell line (CSUASOi006-A-2) from a retinitis pigmentosa patient using CRISPR/Cas9 technology

Retinitis pigmentosa (RP) is a heterogeneous group of hereditary eye disorders characterized by a progressive degeneration of the light-sensing photoreceptor cells in the retina. Currently, there are no effective treatments. In a previous study, we generated a human induced pluripotent stem (iPS) cell line (CSUASOi006-A) from an RP patient carrying a PRPF8 (c.C5792T) mutation. In this study, we corrected the c.5792C > T mutation in the PRPF8 gene using CRISPR/Cas9 technology and generated an isogenic control cell line (CSUASOi006-A-2). This provides an important cellular resource for RP research.

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Publication Details

Journal
Stem Cell Research
Published
2025-04-26
DOI
https://doi.org/10.1016/j.scr.2025.103727
Primary Topic
Retinal Development and Disorders
Type
article
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article

Generation of a gene-corrected isogenic human iPS cell line (CSUASOi006-A-2) from a retinitis pigmentosa patient using CRISPR/Cas9 technology

Xihao Sun, Ruting Zhang, Yuqin Liang, Zekai Cui et al.
Stem Cell Research
Retinal Development and Disorders
article

Generation of a gene-corrected isogenic human iPS cell line (CSUASOi006-A-2) from a retinitis pigmentosa patient using CRISPR/Cas9 technology

Xihao Sun, Ruting Zhang, Yuqin Liang, Zekai Cui, Jiansu Chen, Yuexi Chen, Yuan Liang, Xiaoxue Li, Hang Chen, Wen‐Wei Li, Jianing Gu, Chengcheng Ding, Chunwen Duan
article en

Abstract

Retinitis pigmentosa (RP) is a heterogeneous group of hereditary eye disorders characterized by a progressive degeneration of the light-sensing photoreceptor cells in the retina. Currently, there are no effective treatments. In a previous study, we generated a human induced pluripotent stem (iPS) cell line (CSUASOi006-A) from an RP patient carrying a PRPF8 (c.C5792T) mutation. In this study, we corrected the c.5792C > T mutation in the PRPF8 gene using CRISPR/Cas9 technology and generated an isogenic control cell line (CSUASOi006-A-2). This provides an important cellular resource for RP research.

Stem Cell ResearchVol. 86
Central South University (CN), Shandong Eye Hospital (CN), Xiangya Hospital Central South University (CN), First Affiliated Hospital of Jinan University (CN)
Zero hunger
Openalex Percentile: Top 92%
Retinal Development and Disorders
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Generation of a gene-corrected isogenic human iPS cell line (CSUASOi006-A-2) from a retinitis pigmentosa patient using CRISPR/Cas9 technology — Xihao Sun, Ruting Zhang, et al. · Stem Cell Research (2025) | TGRS Research Map | TGRS