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Journal article : Review

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

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.
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.3390/genes17030283

Authors

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Institution:
University of Oxford
Role:
Author
More by this author
Institution:
University of Oxford
Role:
Author
ORCID:
0000-0002-3096-4682


Publisher:
MDPI
Journal:
Genes More from this journal
Volume:
17
Issue:
3
Pages:
283
Article number:
283
Publication date:
2026-02-27
Acceptance date:
2026-02-24
DOI:
EISSN:
2073-4425
ISSN:
2073-4425


Language:
English
Keywords:
Subtype:
Review
Pubs id:
2390823
Local pid:
pubs:2390823
Source identifiers:
3834422
Deposit date:
2026-03-09
ARK identifier:
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