Bekhite Mohamed M, Hübner Sascha, Kretzschmar Tom, Backsch Claudia, Weise Anja, Klein Elisabeth, Bogoviku Juergen, Westphal Julian, Christian Schulze P
Department of Internal Medicine I, Division of Cardiology, University Hospital Jena, FSU Jena, Germany.
Department of Internal Medicine I, Division of Cardiology, University Hospital Jena, FSU Jena, Germany.
Stem Cell Res. 2024 Dec;81:103565. doi: 10.1016/j.scr.2024.103565. Epub 2024 Sep 21.
One of the main signs we do not know enough about arrhythmogenic right ventricular dysplasia 9/cardiomyopathy (ARVCD9, OMIM #609040, autosomal dominant) is the lack of early markers and therapeutic alternatives. To better study disease pathways in vitro, we generated human induced pluripotent stem cell (hiPSC) lines from the father (UKJi006-A) and son (UKJi001-A), who both shared the same heterozygous mutation in the PKP2 gene (OMIM *602861). While the father had a clinical diagnosis of ARVC, the son lacked the ARVC phenotype. To generate hiPSC lines, non-integrating Sendai virus (SeV) vectors expressing the reprogramming factors (OCT4, SOX2, KLF4, and c-MYC) were used for reprogramming patient peripheral blood mononuclear cells (PBMCs).
我们对致心律失常性右心室发育不良9型/心肌病(ARVCD9,OMIM #609040,常染色体显性遗传)了解不足的主要迹象之一,是缺乏早期标志物和治疗方法。为了在体外更好地研究疾病途径,我们从父亲(UKJi006 - A)和儿子(UKJi001 - A)身上获取了人类诱导多能干细胞(hiPSC)系,他们在PKP2基因(OMIM *602861)上都有相同的杂合突变。父亲被临床诊断为致心律失常性右心室心肌病(ARVC),而儿子没有ARVC表型。为了生成hiPSC系,使用表达重编程因子(OCT4、SOX2、KLF4和c - MYC)的非整合仙台病毒(SeV)载体对患者外周血单核细胞(PBMC)进行重编程。