Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration

被引:641
作者
Mandai, M. [1 ,2 ]
Watanabe, A. [6 ,7 ]
Kurimoto, Y. [1 ,3 ]
Hirami, Y. [1 ,3 ]
Morinaga, C. [2 ]
Daimon, T. [9 ]
Fujihara, M. [1 ,3 ]
Akimaru, H. [2 ,10 ]
Sakai, N. [2 ]
Shibata, Y. [2 ]
Terada, M. [2 ]
Nomiya, Y. [6 ]
Tanishima, S. [11 ]
Nakamura, M. [6 ]
Kamao, H. [2 ,12 ]
Sugita, S. [2 ]
Onishi, A. [2 ]
Ito, T. [4 ]
Fujita, K. [4 ]
Kawamata, S. [5 ]
Go, M. J. [5 ]
Shinohara, C. [13 ]
Hata, K. [13 ]
Sawada, M. [14 ]
Yamamoto, M. [1 ,2 ]
Ohta, S. [1 ]
Ohara, Y. [11 ]
Yoshida, K. [8 ]
Kuwahara, J. [6 ]
Kitano, Y. [6 ]
Amano, N. [6 ]
Umekage, M. [6 ]
Kitaoka, F. [6 ]
Tanaka, A. [6 ]
Okada, C. [11 ]
Takasu, N. [6 ]
Ogawa, S. [8 ]
Yamanaka, S. [6 ,15 ]
Takahashi, M. [1 ,2 ]
机构
[1] Fdn Biomed Res & Innovat, Div Ophthalmol, Inst Biomed Res & Innovat Hosp, Kobe, Hyogo, Japan
[2] RIKEN, Ctr Dev Biol, Lab Retinal Regenerat, Kobe, Hyogo, Japan
[3] Gen Hosp, Kobe City Med Ctr, Dept Ophthalmol, Kobe, Hyogo, Japan
[4] Fdn Biomed Res & Innovat, Inst Biomed Res & Innovat Hosp, Dept Clin Trial Management, Kobe, Hyogo, Japan
[5] Fdn Biomed Res & Innovat, Res & Dev Ctr Cell Therapy, Kobe, Hyogo, Japan
[6] Kyoto Univ, Ctr iPS Cell Res & Applicat, Kyoto, Japan
[7] Kyoto Univ, Inst Integrated Cell Mat Sci iCeMS, Kyoto, Japan
[8] Kyoto Univ, Dept Pathol & Tumor Biol, Grad Sch Med, Kyoto, Japan
[9] Hyogo Coll Med, Dept Biostat, Nishinomiya, Hyogo, Japan
[10] Natl Inst Biomed Innovat Hlth & Nutr, Platform Therapeut Rare Dis, Ibaraki, Japan
[11] Mitsubishi Space Software, Tokyo, Japan
[12] Kawasaki Med Sch, Dept Ophthalmol, Kurashiki, Okayama, Japan
[13] Japan Tissue Engn, Gamagori, Japan
[14] HEALIOS KK, Tokyo, Japan
[15] Gladstone Inst Cardiovasc Dis, San Francisco, CA USA
关键词
AGE-RELATED MACULOPATHY; PIGMENT EPITHELIUM; TRANSPLANTATION; PREVALENCE; TRANSLOCATION; RPE; RANIBIZUMAB; SURGERY; SHEETS;
D O I
10.1056/NEJMoa1608368
中图分类号
R5 [内科学];
学科分类号
100201 [内科学];
摘要
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 agerelated 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.)
引用
收藏
页码:1038 / 1046
页数:9
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