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利用 CRISPR/Cas9 基因编辑技术生成杂合型 COL2A1(p.G1113C) 软骨发育不全突变 iPSC 系,MCRIi019-A-7。

Generation of a heterozygous COL2A1 (p.G1113C) hypochondrogenesis mutation iPSC line, MCRIi019-A-7, using CRISPR/Cas9 gene editing.

机构信息

Murdoch Children's Research Institute, Australia; Department of Paediatrics, University of Melbourne, Australia.

Murdoch Children's Research Institute, Australia; Department of Paediatrics, University of Melbourne, Australia.

出版信息

Stem Cell Res. 2021 Oct;56:102515. doi: 10.1016/j.scr.2021.102515. Epub 2021 Aug 25.

Abstract

The human inherited cartilage disease, Hypochondrogenesis, is caused by mutations in the collagen type II gene, COL2A1. To produce an in vitro disease model, we generated a heterozygous patient mutation, COL2A1 p.G1113C, in an established control human induced pluripotent stem cell (iPSC) line, MCRIi019-A, using CRISPR-Cas9 gene editing. The gene-edited mutant line, MCRIi019-A-7, exhibited normal iPSC characteristics, including normal cell morphology, expression of pluripotency markers, the ability to differentiate into three embryonic germ layers, and normal karyotype. Together with its parental isogenic control, this cell line will be useful for Hypochondrogenesis disease modelling and drug testing.

摘要

人类遗传性软骨病,软骨发育不全症,是由Ⅱ型胶原基因(COL2A1)突变引起的。为了建立体外疾病模型,我们使用 CRISPR-Cas9 基因编辑技术,在已建立的对照人诱导多能干细胞(iPSC)系 MCRIi019-A 中产生了一个杂合的患者突变,COL2A1 p.G1113C。基因编辑突变体系 MCRIi019-A-7 表现出正常的 iPSC 特征,包括正常的细胞形态、多能性标志物的表达、分化为三个胚胎 germ 层的能力以及正常的核型。与它的亲本同基因对照系一起,该细胞系将有助于软骨发育不全症的疾病建模和药物测试。

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