自体诱导干细胞衍生的视网膜细胞治疗黄斑变性。
Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration.
机构信息
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.).
出版信息
N Engl J Med. 2017 Mar 16;376(11):1038-1046. doi: 10.1056/NEJMoa1608368.
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 .).
我们评估了将诱导多能干细胞（iPSC）分化的视网膜色素上皮（RPE）细胞片移植到患有新生血管性年龄相关性黄斑变性的患者中的可行性。iPSC 是从两名晚期新生血管性年龄相关性黄斑变性患者的皮肤成纤维细胞中产生的，并分化为 RPE 细胞。对 RPE 细胞及其衍生的 iPSC 进行了广泛的测试。对其中一名患者进行了包括去除新生血管膜和在视网膜下移植自体 iPSC 衍生的 RPE 细胞片的手术。手术后 1 年，移植的细胞片保持完整，最佳矫正视力没有改善或恶化，并且存在囊样黄斑水肿。（由再生医学实现高速公路计划等资助；大学医院医疗信息网络临床试验注册[UMIN-CTR]编号，UMIN000011929.）。
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