Molecular biotechnologyMay 31, 2021

Using CRISPR Gene Editing as a New Treatment for Eye Diseases

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 14, 2026

Abstract

CRISPR-Cas9 gene-editing has shown potential to improve vision in patients with severe visual loss due to retinal diseases.

Retinal diseases are linked to genetic mutations and can lead to severe visual defects or irreversible blindness.
Gene-editing therapy, particularly through CRISPR-Cas9, could modify abnormal genes associated with these conditions.
CRISPR technology allows for various genetic modifications, including gene deletion, insertion, and expression alteration.
Recent experiments with CRISPR genome surgery have demonstrated improvements in vision for affected patients.
Applications of CRISPR-Cas9 have been explored in conditions such as retinitis pigmentosa, Leber congenital amaurosis, and age-related macular degeneration.
Challenges and limitations of CRISPR technology for clinical use in treating retinal diseases are discussed.

Simplified

Retinal diseases are the primary reasons for severe visual defects and irreversible blindness. Retinal diseases are also inherited and acquired. Both of them are caused by mutations in genes or disruptions in specific gene expression, which can be treated by gene-editing therapy. Clustered regularly interspaced short palindromic repeats (CRISPR-Cas9) system is a frontier of gene-editing tools with great potential for therapeutic applications in the ophthalmology field to modify abnormal genes and treat the genome or epigenome-related retinal diseases. The CRISPR system is able to edit and trim the gene include deletion, insertion, inhibition, activation, replacing, remodeling, epigenetic alteration, and modify the gene expression. CRISPR-based genome editing techniques have indicated the enormous potential to treat retinal diseases that previous treatment was not available for them. Also, recent CRISPR genome surgery experiments have shown the improvement of patient's vision who suffered from severe visual loss. In this article, we review the applications of the CRISPR-Cas9 system in human or animal models for treating retinal diseases such as retinitis pigmentosa (RP), Leber congenital amaurosis (LCA), age-related macular degeneration (AMD), proliferative diabetic retinopathy (PDR), and proliferative vitreoretinopathy (PVR), then we survey limitations of CRISPR system for clinical therapy.

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Using CRISPR Gene Editing as a New Treatment for Eye Diseases

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