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Updated: Feb 4, 2026

Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells
Published on: May 7, 2020
Chemical cocktails enable hepatic reprogramming of human urine-derived cells with a single transcription factor
Wei Tang1,2, Ren Guo2,3, Shi-Jun Shen1
1Shanghai Key Laboratory of Signaling and Disease Research, Laboratory of Receptor-Based Biomedicine, School of Life Sciences and Technology, Tongji University, Shanghai, 200092, China.
Abstract:
Human liver or hepatocyte transplantation is limited by a severe shortage of donor organs. Direct reprogramming of other adult cells into hepatic cells may offer a solution to this problem. In a previous study, we have generated hepatocyte-like cells from mouse fibroblasts using only one transcription factor (TF) plus a chemical cocktail. Here, we show that human urine-derived epithelial-like cells (hUCs) can also be transdifferentiated into human hepatocyte-like cells (hiHeps) using one TF (Foxa3, Hnf1α, or Hnf4α) plus the same chemical cocktail CRVPTD (C, CHIR99021; R, RepSox; V, VPA; P, Parnate; T, TTNPB; and D, Dznep). These hiHeps express multiple hepatocyte-specific genes and display functions characteristic of mature hepatocytes. With the introduction of the large T antigen, these hiHeps can be expanded in vitro and can restore liver function in mice with concanavalin-A-induced acute liver failure. Our study provides a strategy to generate functional hepatocyte-like cells from hUCs by using a single TF plus a chemical cocktail.
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