Recent progress in CRISPR/Cas9 system for eye disorders

Prog Mol Biol Transl Sci. 2025:210:21-46. doi: 10.1016/bs.pmbts.2024.07.018. Epub 2024 Aug 17.

Abstract

Ocular disorders encompass a broad spectrum of phenotypic and clinical symptoms resulting from several genetic variants and environmental factors. The unique anatomy and physiology of the eye facilitate validation of cutting-edge gene editing treatments. Genome editing developments have allowed researchers to treat a variety of diseases, including ocular disorders. The clustered regularly interspaced short palindromic repeats (CRISPR/Cas9) system holds considerable promise for therapeutic applications in the field of ophthalmology, including repair of aberrant genes and treatment of retinal illnesses related to the genome or epigenome. Application of CRISPR/Cas9 systems to the study of ocular disease and visual sciences have yielded innovations including correction of harmful mutations in patient-derived cells and gene modifications in several mammalian models of eye development and disease. In this study, we discuss the generation of several ocular disease models in mammalian cell lines and in vivo systems using a CRISPR/Cas9 system. We also provide an overview of current uses of CRISPR/Cas9 technologies for the treatment of ocular pathologies, as well as future challenges.

Keywords: Blindness; CRISPR/Cas9; Clinical trials; Eye disease; Gene editing; Nucleases; Pathology; Retinal degeneration; Single guide RNA; Therapeutics; Vision impairment.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems* / genetics
  • Eye Diseases* / genetics
  • Eye Diseases* / therapy
  • Gene Editing
  • Humans