Human pluripotent stem cells: A toolbox to understand and treat retinal degeneration

Mol Cell Neurosci. 2020 Sep:107:103523. doi: 10.1016/j.mcn.2020.103523. Epub 2020 Jul 4.

Abstract

Age-related Macular Degeneration (AMD) and Retinitis Pigmentosa (RP) are retinal degenerative disorders that dramatically damage the retina. As there is no therapeutic option for the majority of patients, vision is progressively and irremediably lost. Owing to their unlimited renewal and potency to give rise to any cell type of the human adult body, human pluripotent stem cells (hPSCs) have been extensively studied in recent years to develop more physiologically relevant in vitro cellular models. Such models open new perspectives to investigate the pathological molecular mechanisms of AMD and RP but also in drug screening. Moreover, proof-of-concept of hPSC-derived retinal cell therapy in animal models have led to first clinical trials. This review outlines the recent advances in the use of hPSCs in pathological modeling of retinal degeneration and their use in regenerative medicine. We also address the associated limitations and challenges that need to be overcome when using hPSCs.

Keywords: Age-related Macular Degeneration; Cell therapy; Disease modeling; Human pluripotent stem cells; Retinitis Pigmentosa; Retinopathies.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell Differentiation / physiology
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Pluripotent Stem Cells / cytology*
  • Retina / cytology*
  • Retinal Degeneration / pathology
  • Retinal Degeneration / therapy*
  • Stem Cell Transplantation / methods