Molecular aspects of medicineFeb 17, 2026

Using CRISPR gene editing to treat inherited eye diseases: progress, challenges, and future possibilities

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Feb 18, 2026

Abstract

Recent advances in CRISPR technologies may offer new hope for treating inherited retinal diseases (IRDs).

Over 320 genes are associated with inherited retinal diseases, contributing to their genetic diversity and phenotypic variability.
CRISPR strategies, including gene knockout, exon skipping, and RNA editing, are being explored in preclinical models of IRDs.
Allele-specific targeting and mutation-independent strategies may address both dominant and recessive forms of IRDs.
The first human trial using CRISPR gene editing for CEP290-associated Leber congenital amaurosis has been initiated.
Challenges such as delivery constraints, immune responses, and off-target effects are critical considerations in CRISPR applications.

Simplified

Inherited retinal diseases (IRDs) are a genetically diverse group of disorders characterized by progressive photoreceptor degeneration, leading to vision loss and blindness. With over 320 associated genes and significant phenotypic variability, effective treatment remains challenging. Recent advances in genome editing, particularly CRISPR/Cas-based technologies, have revolutionized therapeutic approaches by enabling precise and customizable DNA and RNA editing. This review explores the application of various CRISPR strategies-such as gene knockout via non-homologous end joining (NHEJ), exon skipping using dual-sgRNAs, homology-directed repair (HDR), base editing (BE), prime editing (PE), RNA editing with Cas13, and epigenetic modulation through CRISPRa/i-in preclinical models of IRDs. Emphasis is placed on allele-specific targeting, gene-agnostic approaches, and mutation-independent strategies to address dominant and recessive forms of disease. We also highlight recent clinical milestones, including the first human trial using CRISPR gene editing for CEP290-associated Leber congenital amaurosis. Finally, we discuss critical challenges, including delivery constraints, immune responses, and off-target effects, along with emerging solutions such as engineered Cas variants, split-intein systems, and advanced off-target detection methods. Together, these advances underscore the transformative potential of CRISPR technologies in treating IRDs and lay the foundation for future clinical translation.

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Declaration of interest statement The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

PubMed

Using CRISPR gene editing to treat inherited eye diseases: progress, challenges, and future possibilities

Abstract

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Funding

Competing interests

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You found one interesting study. We’ll send the next 7.

What Lands in Your Inbox Each Week:

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