Therapeutic applications of gene editing using CRISPR-Cas9 in the posterior segment: review of the literature

Abstract

CRISPR-Cas gene editing has become increasingly relevant in the treatment of several ophthalmic diseases. Its ability to make precise modifications at the DNA or RNA level has enabled targeted approaches for specific mutations involved in conditions such as Leber congenital amaurosis type 10 (LCA10), certain forms of retinitis pigmentosa, and age-related macular degeneration.

This article provides an organized overview of the biological basis of CRISPR-Cas technology and highlights key advances from preclinical studies and early clinical trials. Technical limitations and ongoing safety challenges are also discussed. Programs such as EDIT-101, EDIT-103, and HG202 stand out as important milestones in the evolution of ocular gene editing.