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Therapeutic Potential of Patient iPSC-Derived iMelanocytes in Autologous Transplantation.

Cell reports (2019-04-11)
Li-Ping Liu, Yu-Mei Li, Ning-Ning Guo, Shu Li, Xiaolong Ma, Yi-Xuan Zhang, Yimeng Gao, Jian-Ling Huang, Dong-Xu Zheng, Lu-Yuan Wang, Hui Xu, Lijian Hui, Yun-Wen Zheng
ANOTACE

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.

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Anti-PAX3 antibody produced in rabbit, Prestige Antibodies® Powered by Atlas Antibodies, affinity isolated antibody, buffered aqueous glycerol solution