[Toward establishment of regenerative cell therapy for retinitis pigmentosa using iPS cell derived retinal sheet]

Nihon Yakurigaku Zasshi. 2020;155(2):93-98. doi: 10.1254/fpj.19124.
[Article in Japanese]

Abstract

Retinitis pigmentosa (RP) is a group of hereditary diseases that involve loss of photoreceptors. There has been no established treatment for RP, and it is now the 2nd leading cause of blindness in Japan. Previous clinical researches using human fetal retina transplantation suggested some functional recovery in vision, but it did not become a standard therapy because of ethical concerns for using fetus tissues. Invention of induced pluripotent stem cells (iPSC) in 2006 and the establishment of retinal organoids induction protocol from ES/iPS cells have paved a way of cell therapy for RP without ethical concerns. Our team has shown that mouse iPSC derived retinas can survive and mature after subretinal transplantation to the end-stage retinal degeneration model mice. Further, human ESC derived retinas survived and matured in retinal degeneration monkey models. Recently, we have established a qualitative and quantitative evaluation tool for photoreceptor synapses, QUANTOS, and showed that photoreceptors in mouse iPSC derived retina can form photoreceptor synapses in a time dependent manner after transplantation. We are now moving toward 1st in human clinical trial using iPSC derived retina for RP.

MeSH terms

  • Animals
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Japan
  • Mice
  • Photoreceptor Cells / cytology
  • Retina*
  • Retinitis Pigmentosa / therapy*
  • Stem Cell Transplantation*