通过CRISPR/Cas9技术产生层析成分A/C基因敲除的人类诱导多能干细胞系（ZJULLi007-A）。

IF 0.8 4区医学 Q4 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Stem cell research Pub Date : 2024-10-15 DOI:10.1016/j.scr.2024.103579

Dandan Liu , Jiaxi Shen , Zongkuai Yang , Hangping Fan , Hao Wang , Ping Liang , Tingyu Gong

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引用次数: 0

摘要

Lamin A/C 是一种由 LMNA 基因编码的蛋白质，属于核薄层蛋白家族。LMNA 基因突变会导致多种疾病：埃默里-德赖福斯肌肉营养不良症、家族性部分脂肪营养不良症、肢腰肌营养不良症、扩张型心肌病、夏科-玛丽-牙病和哈钦森-吉尔福德早衰综合征。本研究利用CRISPR/Cas9基因组编辑技术，成功培育出了lamin A/C基因敲除的人类诱导多能干细胞系，并证实该细胞系具有正常的多能性和核型。

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Generation of a lamin A/C knockout human induced pluripotent stem cell line (ZJULLi007-A) via CRISPR/Cas9

Lamin A/C is a protein encoded by the LMNA gene and belongs to the nuclear lamina protein family. Mutations in the LMNA gene lead to several diseases: Emery-Dreifuss muscular dystrophy, familial partial lipodystrophy, limb girdle muscular dystrophy, dilated cardiomyopathy, Charcot-Marie-Tooth disease, and Hutchinson-Gilford progeria syndrome. In this study, a lamin A/C knockout human induced pluripotent stem cell line was successfully generated using the CRISPR/Cas9 genome-editing technology, which was confirmed with normal pluripotency and karyotype.

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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.