N Engl J Med. 2017 Mar 16;376(11):1038-1046. doi: 10.1056/NEJMoa1608368.

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

Mandai M¹, Watanabe A¹, Kurimoto Y¹, Hirami Y¹, Morinaga C¹, Daimon T¹, Fujihara M¹, Akimaru H¹, Sakai N¹, Shibata Y¹, Terada M¹, Nomiya Y¹, Tanishima S¹, Nakamura M¹, Kamao H¹, Sugita S¹, Onishi A¹, Ito T¹, Fujita K¹, Kawamata S¹, Go MJ¹, Shinohara C¹, Hata KI¹, Sawada M¹, Yamamoto M¹, Ohta S¹, Ohara Y¹, Yoshida K¹, Kuwahara J¹, Kitano Y¹, Amano N¹, Umekage M¹, Kitaoka F¹, Tanaka A¹, Okada C¹, Takasu N¹, Ogawa S¹, Yamanaka S¹, Takahashi M¹.

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 .).