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Gene therapy in the anterior eye segment

Author(s):

Cynthia Amador, Ruchi Shah, Sean Ghiam, Andrei A. Kramerov and Alexander V. Ljubimov*  

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.

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.

Affiliation:

Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA, Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA, Sackler School of Medicine, New York State/American Program of Tel Aviv University, Tel Aviv, Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA, Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA



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