肯尼迪病AR突变患者诱导多能干细胞系的生成

IF 0.8 4区医学 Q4 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Stem cell research Pub Date : 2024-12-30 DOI:10.1016/j.scr.2024.103649

Meng Zhang, Rui Liu, Pengpeng Sun, Chengsen Zhang, Jingdong Wang, Changjiang Li

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引用次数: 0

摘要

从携带AR基因CAG重复扩增突变的肯尼迪病（KD）患者获得了人诱导多能干细胞（iPSC）系。外周血单个核细胞（PBMCs）通过非整合递送KFL4、OCT4、SOX2、BCL-XL和c-MYC进行重编程。iPSC细胞系表达多能性标记，显示正常核型，并能在体外分化为三种胚层。该iPSC系为研究人类KD提供了一种有价值的细胞模型。

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Generation of an induced pluripotent stem cell line from a Kennedy Disease patient with AR mutation.

A human induced pluripotent stem cell (iPSC) line was generated from patient with Kennedy Disease (KD), who carried the CAG repeat expansion mutation in AR gene. Peripheral blood mononuclear cells (PBMCs) were reprogrammed using non-integrating delivery of KFL4, OCT4, SOX2, BCL-XL and c-MYC. The iPSC line expresses pluripotency markers, displays a normal karyotype, and is capable of differentiate into three germ layers in vitro. This iPSC line represents a valuable cell model for studing KD in humans.

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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.