利用CRISPR/Cas9技术从视网膜色素变性患者身上获得基因校正的等基因人类iPS细胞系(CSUASOi006-A-2

IF 0.8 4区医学 Q4 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Stem cell research Pub Date : 2025-04-26 DOI:10.1016/j.scr.2025.103727

Hang Chen , Yuqin Liang , Yuan Liang , Yuexi Chen , Xiaoxue Li , Ruting Zhang , Chunwen Duan , Wenwei Li , Zekai Cui , Jianing Gu , Chengcheng Ding , Xihao Sun , Jiansu Chen

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引用次数: 0

摘要

色素性视网膜炎（RP）是一种异质性的遗传性眼部疾病，其特征是视网膜中感光细胞的进行性变性。目前还没有有效的治疗方法。在之前的研究中，我们从一名携带PRPF8 （c.c c5792t）突变的RP患者身上获得了一株人诱导多能干细胞（CSUASOi006-A）。在本研究中，我们修正了c.5792C >；利用CRISPR/Cas9技术进行PRPF8基因T突变，生成等基因对照细胞系（CSUASOi006-A-2）。这为RP研究提供了重要的细胞资源。

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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

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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来源期刊

Stem cell research 生物-生物工程与应用微生物

CiteScore

2.20

自引率

8.30%

发文量

338

审稿时长

55 days

期刊介绍： Stem Cell Research is dedicated to publishing high-quality manuscripts focusing on the biology and applications of stem cell research. Submissions to Stem Cell Research, may cover all aspects of stem cells, including embryonic stem cells, tissue-specific stem cells, cancer stem cells, developmental studies, stem cell genomes, and translational research. Stem Cell Research publishes 6 issues a year.