Zhu Jingting, Guo Gongbo, Mehryab Fatemeh, McCulloch Mary Kate, Junior Wilson Marques, Shy Michael E, Hester Mark E, Rashnonejad Afrooz
Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, 575 Children's Crossroad, Columbus, OH 43215, USA.
Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital, 575 Children's Crossroad, Columbus, OH 43215, USA; Molecular, Cellular, and Developmental Biology Program, The Ohio State University, Columbus, OH 43210, USA.
Stem Cell Res. 2025 Apr;84:103684. doi: 10.1016/j.scr.2025.103684. Epub 2025 Feb 15.
Charcot-Marie-Tooth type 1B (CMT1B) is a demyelination neuropathy caused by over 200 mutations in the myelin protein zero (MPZ) gene. Here, we generated two induced pluripotent stem cell (iPSC) lines from fibroblasts isolated from the skin biopsies of CMT1B patients, each carrying a distinct MPZ mutation (Arg98Cys and Ser63del). The iPSC lines created in this work retained their respective MPZ mutation, exhibited normal karyotypes, expressed pluripotency markers, and demonstrated the ability to differentiate into three germ-layer cell types. These lines offer a valuable tool for exploring and modeling dominant CMT1B disease within a human cellular framework.
1B型夏科-马里-图思病(CMT1B)是一种脱髓鞘性神经病变,由髓磷脂蛋白零(MPZ)基因中的200多种突变引起。在此,我们从CMT1B患者皮肤活检分离出的成纤维细胞中生成了两条诱导多能干细胞(iPSC)系,每条携带一个不同的MPZ突变(Arg98Cys和Ser63del)。本研究中创建的iPSC系保留了各自的MPZ突变,表现出正常的核型,表达多能性标志物,并展示了分化为三种胚层细胞类型的能力。这些细胞系为在人类细胞框架内探索和模拟显性CMT1B疾病提供了有价值的工具。