# Precision Is Not Enough: When Tools Outpace Translation in Ocular Gene Therapy

**Authors:** Maram E. A. Abdalla Elsayed, Robert E. MacLaren

PMC · DOI: 10.3390/genes17030283 · Genes · 2026-02-27

## TL;DR

The paper discusses how advances in gene therapy for eye diseases are outpacing their practical application in medicine.

## Contribution

The paper provides a critical evaluation of current gene therapy strategies and translational challenges in ocular gene therapy.

## Key findings

- The eye is a promising platform for gene therapy due to its accessibility and immune privilege.
- CRISPR and genome-editing technologies face significant limitations in ocular applications.
- Translational challenges remain a barrier to integrating genetic therapies into routine ophthalmic practice.

## Abstract

Advances in molecular biology have positioned the eye as a leading platform for gene therapy, owing to its surgical accessibility, relative immune privilege, and the ability of the contralateral eye to serve as an anatomical control. We trace the historical evolution of gene discovery, synthesize current gene therapy strategies for inherited and acquired ocular disorders, critically evaluating the limitations of CRISPR and related genome-editing technologies, and examine the key scientific and translational challenges that must be addressed for genetic therapies to be integrated into routine ophthalmic practice.

## Full-text entities

- **Diseases:** inherited and acquired ocular disorders (MESH:D025861)

## Full text

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## Figures

3 figures with captions in the complete paper: https://tomesphere.com/paper/PMC13026294/full.md

## References

155 references — full list in the complete paper: https://tomesphere.com/paper/PMC13026294/full.md

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Source: https://tomesphere.com/paper/PMC13026294