Alowaysi Maryam, Fiacco Elisabetta, Astro Veronica, Adamo Antonio
Biological and Environmental Science and Engineering Division, King Abdullah University of Science and Technology, Thuwal 23955-6900, Saudi Arabia.
Biological and Environmental Science and Engineering Division, King Abdullah University of Science and Technology, Thuwal 23955-6900, Saudi Arabia.
Stem Cell Res. 2020 Dec;49:102008. doi: 10.1016/j.scr.2020.102008. Epub 2020 Sep 22.
Klinefelter Syndrome (KS) is the most frequent X chromosome aneuploidy in males. KS patients with 47-XXY, 48-XXXY and 49-XXXXY karyotypes endure inter-individual phenotypic variabilities including infertility, cardiac diseases, metabolic and psychiatric disorders. We derived iPSC lines from a high-grade 49-XXXXY KS and two healthy donors' fibroblasts. Importantly, the healthy controls XY and XX are direct relatives to KS patients, thus enabling functional comparisons of healthy and disease iPSCs with partially matched genetic backgrounds. These iPSC lines provide an unprecedented cellular tool to study KS pathophysiology at the pluripotent stage as well as during differentiation into disease relevant cell types.
克兰费尔特综合征(KS)是男性中最常见的X染色体非整倍体疾病。核型为47-XXY、48-XXXY和49-XXXXY的KS患者存在个体间的表型差异,包括不孕、心脏病、代谢和精神疾病。我们从一名核型为49-XXXXY的重度KS患者以及两名健康供体的成纤维细胞中获得了诱导多能干细胞(iPSC)系。重要的是,健康对照XY和XX是KS患者的直系亲属,因此能够对具有部分匹配遗传背景的健康和疾病iPSC进行功能比较。这些iPSC系为研究KS在多能阶段以及分化为疾病相关细胞类型过程中的病理生理学提供了前所未有的细胞工具。