Human retinal progenitor cell transplantation preserves vision

J Biol Chem. 2014 Mar 7;289(10):6362-6371. doi: 10.1074/jbc.M113.513713. Epub 2014 Jan 9.


Cell transplantation is a potential therapeutic strategy for retinal degenerative diseases involving the loss of photoreceptors. However, it faces challenges to clinical translation due to safety concerns and a limited supply of cells. Human retinal progenitor cells (hRPCs) from fetal neural retina are expandable in vitro and maintain an undifferentiated state. This study aimed to investigate the therapeutic potential of hRPCs transplanted into a Royal College of Surgeons (RCS) rat model of retinal degeneration. At 12 weeks, optokinetic response showed that hRPC-grafted eyes had significantly superior visual acuity compared with vehicle-treated eyes. Histological evaluation of outer nuclear layer (ONL) characteristics such as ONL thickness, spread distance, and cell count demonstrated a significantly greater preservation of the ONL in hRPC-treated eyes compared with both vehicle-treated and control eyes. The transplanted hRPCs arrested visual decline over time in the RCS rat and rescued retinal morphology, demonstrating their potential as a therapy for retinal diseases. We suggest that the preservation of visual acuity was likely achieved through host photoreceptor rescue. We found that hRPC transplantation into the subretinal space of RCS rats was well tolerated, with no adverse effects such as tumor formation noted at 12 weeks after treatment.

Keywords: Eye; Neuroprogenitor Cell; Retinal Degeneration; Retinal Progenitor Cells; Stem Cells; Transplantation.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Separation
  • Cells, Cultured
  • Disease Models, Animal
  • Embryonic Stem Cells / transplantation*
  • Fetus / cytology
  • Humans
  • Pigment Epithelium of Eye / transplantation*
  • Rats
  • Retina* / cytology
  • Retina* / embryology
  • Retina* / physiology
  • Retinal Degeneration / physiopathology
  • Retinal Degeneration / surgery*
  • Stem Cell Transplantation*
  • Visual Acuity