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Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration.
N Engl J Med. 2017 03 16; 376(11):1038-1046.NEJM

Abstract

We assessed the feasibility of transplanting a sheet of retinal pigment epithelial (RPE) cells differentiated from induced pluripotent stem cells (iPSCs) in a patient with neovascular age-related macular degeneration. The iPSCs were generated from skin fibroblasts obtained from two patients with advanced neovascular age-related macular degeneration and were differentiated into RPE cells. The RPE cells and the iPSCs from which they were derived were subject to extensive testing. A surgery that included the removal of the neovascular membrane and transplantation of the autologous iPSC-derived RPE cell sheet under the retina was performed in one of the patients. At 1 year after surgery, the transplanted sheet remained intact, best corrected visual acuity had not improved or worsened, and cystoid macular edema was present. (Funded by Highway Program for Realization of Regenerative Medicine and others; University Hospital Medical Information Network Clinical Trials Registry [UMIN-CTR] number, UMIN000011929 .).

Authors+Show Affiliations

From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).From the Division of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (M.M., Y. Kurimoto, Y.H., M.F., M.Y., S. Ohta, M. Takahashi), Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology (M.M., C.M., H.A., N.S., Y.S., M. Terada, H.K., S.S., A.O., M.Y., M. Takahashi), the Department of Ophthalmology, Kobe City Medical Center General Hospital (Y.Kurimoto, Y.H., M.F.), the Department of Clinical Trial Management, Institute of Biomedical Research and Innovation Hospital, Foundation for Biomedical Research and Innovation (T.I., K.F.), and Research and Development Center for Cell Therapy, Foundation for Biomedical Research and Innovation (S.K., M.J.G.), Kobe, Center for iPS Cell Research and Application (CiRA) (A.W., Y.N., M.N., J.K., Y. Kitano, N.A., M.U., F.K., A.T., N.T., S.Y.), Institute for Integrated Cell-Material Sciences (iCeMS) (A.W.), and Department of Pathology and Tumor Biology, Graduate School of Medicine (K.Y., S. Ogawa), Kyoto University, Kyoto, the Department of Biostatistics, Hyogo College of Medicine, Nishinomiya (T.D.), Platform of Therapeutics for Rare Disease, National Institutes of Biomedical Innovation, Health, and Nutrition, Ibaraki (H.A.), Mitsubishi Space Software, Tokyo (S.T., Y.O., C.O.), the Department of Ophthalmology, Kawasaki Medical School, Kurashiki (H.K.), Japan Tissue Engineering, Gamagori (C.S., K.H.), and HEALIOS K.K., Tokyo (M.S.) - all in Japan; and Gladstone Institute of Cardiovascular Disease, San Francisco (S.Y.).

Pub Type(s)

Case Reports
Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

28296613

Citation

Mandai, Michiko, et al. "Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration." The New England Journal of Medicine, vol. 376, no. 11, 2017, pp. 1038-1046.
Mandai M, Watanabe A, Kurimoto Y, et al. Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. N Engl J Med. 2017;376(11):1038-1046.
Mandai, M., Watanabe, A., Kurimoto, Y., Hirami, Y., Morinaga, C., Daimon, T., Fujihara, M., Akimaru, H., Sakai, N., Shibata, Y., Terada, M., Nomiya, Y., Tanishima, S., Nakamura, M., Kamao, H., Sugita, S., Onishi, A., Ito, T., Fujita, K., ... Takahashi, M. (2017). Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. The New England Journal of Medicine, 376(11), 1038-1046. https://doi.org/10.1056/NEJMoa1608368
Mandai M, et al. Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. N Engl J Med. 2017 03 16;376(11):1038-1046. PubMed PMID: 28296613.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. AU - Mandai,Michiko, AU - Watanabe,Akira, AU - Kurimoto,Yasuo, AU - Hirami,Yasuhiko, AU - Morinaga,Chikako, AU - Daimon,Takashi, AU - Fujihara,Masashi, AU - Akimaru,Hiroshi, AU - Sakai,Noriko, AU - Shibata,Yumiko, AU - Terada,Motoki, AU - Nomiya,Yui, AU - Tanishima,Shigeki, AU - Nakamura,Masahiro, AU - Kamao,Hiroyuki, AU - Sugita,Sunao, AU - Onishi,Akishi, AU - Ito,Tomoko, AU - Fujita,Kanako, AU - Kawamata,Shin, AU - Go,Masahiro J, AU - Shinohara,Chikara, AU - Hata,Ken-Ichiro, AU - Sawada,Masanori, AU - Yamamoto,Midori, AU - Ohta,Sachiko, AU - Ohara,Yasuo, AU - Yoshida,Kenichi, AU - Kuwahara,Junko, AU - Kitano,Yuko, AU - Amano,Naoki, AU - Umekage,Masafumi, AU - Kitaoka,Fumiyo, AU - Tanaka,Azusa, AU - Okada,Chihiro, AU - Takasu,Naoko, AU - Ogawa,Seishi, AU - Yamanaka,Shinya, AU - Takahashi,Masayo, PY - 2017/3/16/entrez PY - 2017/3/16/pubmed PY - 2017/4/25/medline SP - 1038 EP - 1046 JF - The New England journal of medicine JO - N Engl J Med VL - 376 IS - 11 N2 - We assessed the feasibility of transplanting a sheet of retinal pigment epithelial (RPE) cells differentiated from induced pluripotent stem cells (iPSCs) in a patient with neovascular age-related macular degeneration. The iPSCs were generated from skin fibroblasts obtained from two patients with advanced neovascular age-related macular degeneration and were differentiated into RPE cells. The RPE cells and the iPSCs from which they were derived were subject to extensive testing. A surgery that included the removal of the neovascular membrane and transplantation of the autologous iPSC-derived RPE cell sheet under the retina was performed in one of the patients. At 1 year after surgery, the transplanted sheet remained intact, best corrected visual acuity had not improved or worsened, and cystoid macular edema was present. (Funded by Highway Program for Realization of Regenerative Medicine and others; University Hospital Medical Information Network Clinical Trials Registry [UMIN-CTR] number, UMIN000011929 .). SN - 1533-4406 UR - https://www.unboundmedicine.com/medline/citation/28296613/Autologous_Induced_Stem_Cell_Derived_Retinal_Cells_for_Macular_Degeneration_ L2 - https://www.nejm.org/doi/10.1056/NEJMoa1608368?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -