Center for Regenerative Therapies (BCRT), Berlin Institute of Health at Charité - Universitätsmedizin Berlin, Augustenburger Platz 1, 13353 Berlin, Germany.
Center for Regenerative Therapies (BCRT), Berlin Institute of Health at Charité - Universitätsmedizin Berlin, Augustenburger Platz 1, 13353 Berlin, Germany; Deutsches Herzzentrum der Charité (DHZC), Department of Cardiothoracic and Vascular Surgery, Augustenburger Platz 1, 13353 Berlin, Germany.
Stem Cell Res. 2023 Jun;69:103071. doi: 10.1016/j.scr.2023.103071. Epub 2023 Mar 20.
Autosomal dominant polycystic kidney disease (ADPKD) is a common genetic disorder of adults, characterized by uncontrolled cysts formation that causes a gradual impairment of kidney function. We generated a human induced pluripotent stem cell (hiPSC) line from the urinary cells of a patient diagnosed with ADPKD using a non-integrating Epi5™ Episomal iPSC reprogramming strategy. Characterization of the cell line was performed regarding their undifferentiated status, differentiation potential, and quality control for karyotypic integrity, identity, and clearance of reprogramming vectors. The newly derived hiPSC line, namely BCRTi007-A, can be used in vitro for disease modeling of ADPKD as well as testing for novel therapeutic approaches.
常染色体显性多囊肾病（ADPKD）是一种常见的成人遗传性疾病，其特征是无控制的囊肿形成，导致肾功能逐渐受损。我们使用非整合的 Epi5™ Episomal iPSC 重编程策略，从诊断为 ADPKD 的患者的尿细胞中生成了一个人类诱导多能干细胞（hiPSC）系。对细胞系的未分化状态、分化潜能以及核型完整性、身份和重编程载体清除的质量控制进行了特征描述。新衍生的 hiPSC 系，即 BCRTi007-A，可用于体外 ADPKD 的疾病建模以及新型治疗方法的测试。
