Retinal Organoids: Innovative Tools for Understanding Retinal Degeneration

Int J Mol Sci. 2025 Apr 1;26(7):3263. doi: 10.3390/ijms26073263.

Abstract

Retinal degenerative diseases (RDDs) comprise diverse genetic and phenotypic conditions that cause progressive retinal dysfunction and cell loss, leading to vision impairment or blindness. Most RDDs lack appropriate animal models for their study, which affects understanding their disease mechanisms and delays the progress of new treatment development. Recent advances in stem cell engineering, omics, and organoid technology are facilitating research into diseases for which there are no previously existing models. The development of retinal organoids produced from human stem cells has impacted the study of retinal development as well as the development of in vitro models of diseases, opening possibilities for applications in regenerative medicine, drug discovery, and precision medicine. In this review, we recapitulate research in the retinal organoid models for RDD, mentioning some of the main pathways underlying retinal neurodegeneration that can be studied in these new models, as well as their limitations and future challenges in this rapidly advancing field.

Keywords: photoreceptors; retinal degeneration; retinal organoids; stem cells.

Publication types

  • Review

MeSH terms

  • Animals
  • Humans
  • Organoids* / metabolism
  • Organoids* / pathology
  • Retina* / cytology
  • Retina* / metabolism
  • Retina* / pathology
  • Retinal Degeneration* / metabolism
  • Retinal Degeneration* / pathology