α - 1抗胰蛋白酶缺乏症（AATD）患儿诱导多能干细胞系HHUUKDi013-A （ISRM-AATD-iPSC-3）的生成

IF 0.8 4区医学 Q4 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Stem cell research Pub Date : 2025-06-28 DOI:10.1016/j.scr.2025.103762

Christiane Loerch , Rabea Hokamp , Wasco Wruck , David Katzer , Alexander Weigert , Alexander Machui , Nina Graffmann , Rainer Ganschow , James Adjaye

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引用次数: 0

摘要

从一名男性儿科aatd患者的尿液中分离出具有同源Pi*ZZ基因型的肾祖细胞。six2阳性尿源性肾祖细胞（udrpc）通过表达OCT4、KLF4、c-MYC、SOX2、NANOG和LIN28的episomal质粒的无整合核转染，被重编程为iPSCs。ISRM-AATD-iPSC-3表达多能基因和蛋白，具有胚状体形成能力，携带亲本Pi*ZZ基因型。全球转录组分析显示，人胚胎干细胞H9与ISRM-AATD-iPSC-3的相关系数为0.9091。

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Generation of an induced pluripotent stem cell line HHUUKDi013-A (ISRM-AATD-iPSC-3) from a pediatric patient of Alpha-I Antitrypsin Deficiency (AATD)

Renal progenitor cells were isolated from urine from a male, pediatric AATD-patient, harboring the homologous Pi*ZZ genotype. SIX2-positive urine derived renal progenitor cells (UdRPCs) were reprogrammed to iPSCs by integration-free nucleofection of episomal-based plasmids expressing OCT4, KLF4, c-MYC, SOX2, NANOG and LIN28. ISRM-AATD-iPSC-3 show pluripotent gene and protein expression, are capable of forming embryoid bodies and carry the parental Pi*ZZ genotype. Global transcriptome analyses revealed a correlation coefficient 0.9091 between the human embryonic stem cell line H9 and ISRM-AATD-iPSC-3.

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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.