Preclinical study on induction of pluripotent stem cells from urine of dilated cardiomyopathy patients.

Abstract Dilated cardiomyopathy (DCM) is featured by left or bilateral ventricular dilation combined with systolic dysfunction. Its clinical manifestations include heart enlargement, cardiac failure, arrhythmia and cardiac arrest. Medication and heart transplantation have but only limited treatment effect on DCM. Induced pluripotent stem cell (iPSC) treatment provides a new solution for DCM treatment. Human renal epithelial cells were extracted from the urine of patients with DCM and transfected with Sendai virus carrying OCT3/4, Sox2, Klf4 and c-Myc gene to generate iPSCs by reprogramming. The morphology and pluripotency of iPSCs obtained from the renal epithelial cells from patients with DCM were confirmed, as well as the growth characteristics, immunohistochemical features and surface markers of embryonic stem cells. Teratoma was formed in vivo. We demonstrated that it was feasible to obtain iPSCs from the urine of patients with DCM. This technique lays down the cytological foundation for understanding the pathogenesis and for drug screening and gene therapy for DCM.