The directed differentiation of human-induced pluripotent stem cells (hiPSCs) into clinically useful cell types has been vigorously examined. Transplantation methods and therapeutic potentials of hiPSC-derived cells have also been evaluated using animal disease models. Episomal vectors were successfully used to generate iPSCs without the use of viral vectors, which may cause cancer by damaging the genome. An iPSC stock derived from voluntary HLA homozygous donors is being prepared for use in regenerative therapy. These efforts have led to the start of clinical trials for the transplantation of hiPSC-derived retinal cells in Japan. Further research on reconstruction methods for functional tissues and organs from hiPSCs would resolve the problem of donor shortage in organ transplantation as well as accelerate regenerative medicine.