CRISPR/ cas9介导的纯合子CBR2敲除H1人胚胎干细胞系的产生

IF 0.8 4区医学 Q4 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Stem cell research Pub Date : 2025-02-11 DOI:10.1016/j.scr.2025.103677

Lei Zhang , Fengfeng Zhang , Mingze Yao

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引用次数: 0

摘要

CRB2基因突变可引起多种常染色体隐性遗传病，如leber先天性黑内障、视网膜色素变性、脑室肿大合并囊性肾病等。然而，CRB2在细胞命运决定中的确切作用尚不清楚。在这里，我们使用CRISPR/Cas9系统生成了纯合子CRB2敲除（CRB2−/−）H1人胚胎干细胞（hESCs）。该细胞系保持了正常的形态和核型，表达了多能细胞。重要的是，这种细胞系有能力分化成三个胚层。CRB2−/−hESCs为研究由CRB2突变引起的遗传疾病的机制提供了宝贵的资源。

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CRISPR/Cas9-mediated generation of a homozygous CBR2 knockout H1 human embryonic stem cell line

Mutations in the Crumbs homolog 2 (CRB2) gene cause various autosomal recessive genetic diseases, such as leber congenital amaurosis, retinitis pigmentosa and ventriculomegaly with cystic kidney disease. However, the precise roles of CRB2 in cell fate determination remains unknown. Here, we generated a homozygous CRB2 knockout (CRB2^−/−) H1 human embryonic stem cells (hESCs) using CRISPR/Cas9 system. This cell line maintained a normal morphology and karyotype, and expressed the pluripotency makers. Importantly, the cell line has the ability to differentiate into three germ layers. The CRB2^−/− hESCs provide valuable resources for studying the mechanisms of genetic diseases caused by CRB2 mutations.

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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.