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Current Gene Therapy


ISSN (Print): 1566-5232
ISSN (Online): 1875-5631

Review Article

Gene Therapy in the Anterior Eye Segment

(E-pub Ahead of Print)
Author(s): Cynthia Amador, Ruchi Shah, Sean Ghiam, Andrei A. Kramerov and Alexander V. Ljubimov*

DOI: 10.2174/1566523221666210423084233

open access plus


This review provides comprehensive information about the advances in gene therapy in the anterior segment of the eye, including cornea, conjunctiva, lacrimal gland, and trabecular meshwork. We discuss gene delivery systems, including viral and non-viral vectors as well as gene editing techniques, mainly CRISPR-Cas9, and epigenetic treatments, including antisense and siRNA therapeutics. We also provide a detailed analysis of various anterior segment diseases where gene therapy has been tested with corresponding outcomes. Disease conditions include corneal and conjunctival fibrosis and scarring, corneal epithelial wound healing, corneal graft survival, corneal neovascularization, genetic corneal dystrophies, herpetic keratitis, glaucoma, dry eye disease, and other ocular surface diseases. Although most of the analyzed results on the use and validity of gene therapy at the ocular surface have been obtained in vitro or using animal models, we also discuss the available human studies. Gene therapy approaches are currently considered very promising as emerging future treatments of various diseases, and this field is rapidly expanding.

Keywords: Gene therapy, cornea, corneal dystrophy, corneal wound healing, keratitis, corneal neovascularization, glaucoma, corneal dystrophy, dry eye, graft survival, non-viral vector, nanoconstruct, drug delivery, adenovirus, adeno-associated virus, retrovirus, lentivirus, antisense, siRNA, CRISPR-Cas9.

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