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Updated: Jan 26, 2026

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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Therapeutic Potential of Patient iPSC-Derived iMelanocytes in Autologous Transplantation

Li-Ping Liu1, Yu-Mei Li2, Ning-Ning Guo2

  • 1Institute of Regenerative Medicine, Affiliated Hospital of Jiangsu University, Jiangsu University, Zhenjiang, Jiangsu 212001, China; Department of Dermatology, Affiliated Hospital of Jiangsu University, Jiangsu University, Zhenjiang, Jiangsu 212001, China; Faculty of Medicine, University of Tsukuba, Ibaraki 305-8575, Japan.

Cell Reports
|April 11, 2019
PubMed

Induced pluripotent stem cells (iPSCs) are a promising melanocyte source as they propagate indefinitely and can be established from patients. However, the in vivo functions of human iPSC-derived melanocytes (hiMels) remain unknown. Here, we generated hiMels from vitiligo patients using a three-dimensional system with enhanced differentiation efficiency, which showed characteristics of human epidermal melanocytes with high sequence similarity and involved in multiple vitiligo-associated signaling pathways. A modified hair follicle reconstitution assay in vivo showed that MITF+PAX3+TYRP1+ hiMels were localized in the mouse hair bulb and epidermis and produced melanin up to 7 weeks after transplantation, whereas MITF+PAX3+TYRP1- hiMelanocyte stem cells integrated into the bulge-subbulge regions. Overall, these data demonstrate the long-term functions of hiMels in vivo to reconstitute pigmented hair follicles and to integrate into normal regions for both mature melanocytes and melanocyte stem cells, providing an alternative source of personalized cellular therapy for depigmentation.

Keywords:
3D differentiation systemEBcellular transplantation therapyembryoid bodyhair follicle reconstitution in vivohair follicle stem cellshiMelshuman epidermal melanocyteshuman iPSC-derived melanocytesiPSCsmelanocyte stem cellspatient induced pluripotent stem cellsthree-dimensional differentiation systemvitiligo

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