Wu Kangkang, Wang Chunli, Hu Huiyi, Shen Ying, Zheng Yucan, Hu Guorui, Liu Zhifeng
Department of Infectious Disease, Children's Hospital of Nanjing Medical University, Nanjing 210008, China.
Nanjing Key Laboratory of Pediatrics, Children's Hospital of Nanjing Medical University, Nanjing 210008, China.
Stem Cell Res. 2025 Sep;87:103745. doi: 10.1016/j.scr.2025.103745. Epub 2025 Jun 5.
progressive familial intrahepatic cholestasis 3 (PFIC3) caused by mutations in ABCB4 gene is a rare lethal autosomal recessive liver disease. Here, we detail the reprogramming of peripheral blood mononuclear cells (PBMCs) using non-integrative Sendai virus to produce induced pluripotent stem cells (iPSCs) from a child carrying the ABCB4 mutation. The obtained iPSC presented normal karyotype, high expression of pluripotency markers and the capacity to differentiate into cells of three germ layers.
由ABCB4基因突变引起的进行性家族性肝内胆汁淤积症3型（PFIC3）是一种罕见的致死性常染色体隐性肝病。在此，我们详细介绍了使用非整合型仙台病毒对外周血单个核细胞（PBMC）进行重编程，以从一名携带ABCB4突变的儿童中产生诱导多能干细胞（iPSC）。所获得的iPSC呈现出正常的核型、多能性标志物的高表达以及分化为三个胚层细胞的能力。
