Introduction of an RS1 mutation causative variant consistent with identified XLRS patient using CRISPR/Cas9 technology in normal iPSC

X-linked retinoschisis (XLRS) is a common retinal genetic disease that occurs in juvenile males and causes progressive visual impairment. This presents a schisis in the macula or peripheral retina of bilateral eyes, which has no effective treatment. Here, we introduced the RS1 (c.C304T, p.R102W) mutation into a normal induced pluripotent stem (iPS) cell line using CRISPR/Cas9 technology. This missense mutation was consistent with that observed in the XLRS patient-derived iPS cell line (CSUASOi001-A). Conclusively, establishing a directed gene mutation cell line (CSUi007-A) provides a useful cell resource to investigate XLRS pathogenesis.

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Publication Details

Journal
Stem Cell Research
Published
2024-09-01
DOI
https://doi.org/10.1016/j.scr.2024.103549
Primary Topic
Retinal Development and Disorders
Type
article
Field-Weighted Citation Impact
0.00

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article

Introduction of an RS1 mutation causative variant consistent with identified XLRS patient using CRISPR/Cas9 technology in normal iPSC

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

Introduction of an RS1 mutation causative variant consistent with identified XLRS patient using CRISPR/Cas9 technology in normal iPSC

Xihao Sun, Jiansu Chen, Yuqin Liang, Zekai Cui, Shibo Tang, Shengru Mao, Chunwen Duan, Jianing Gu, Bing Jiang, Chengcheng Ding
article en

Abstract

X-linked retinoschisis (XLRS) is a common retinal genetic disease that occurs in juvenile males and causes progressive visual impairment. This presents a schisis in the macula or peripheral retina of bilateral eyes, which has no effective treatment. Here, we introduced the RS1 (c.C304T, p.R102W) mutation into a normal induced pluripotent stem (iPS) cell line using CRISPR/Cas9 technology. This missense mutation was consistent with that observed in the XLRS patient-derived iPS cell line (CSUASOi001-A). Conclusively, establishing a directed gene mutation cell line (CSUi007-A) provides a useful cell resource to investigate XLRS pathogenesis.

Stem Cell ResearchVol. 81
Central South University (CN), Jinan University (CN), Second Xiangya Hospital of Central South University (CN)
National Natural Science Foundation of China
Openalex Percentile: Top 87%
Retinal Development and Disorders
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