Title:Gene Therapy in the Anterior Eye Segment
Volume: 22
Issue: 2
Author(s): Cynthia Amador, Ruchi Shah, Sean Ghiam, Andrei A. Kramerov and Alexander V. Ljubimov*
Affiliation:
- Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars-
Sinai Medical Center, Los Angeles, CA, USA
- Department of Medicine, David Geffen School of Medicine, University of California
Los Angeles, Los Angeles, CA, USA
Keywords:
Gene therapy, cornea, corneal dystrophy, corneal wound healing, keratitis, corneal neovascularization, glaucoma, dry eye, graft survival, non-viral vector, nanoconstruct, drug delivery, adenovirus, adeno-associated virus, retrovirus, lentivirus, antisense, siRNA, CRISPR-Cas9.
Abstract: 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.
