Gene Therapy in the Anterior Eye Segment

Overview

Journal Curr Gene Ther

Publisher Bentham Science Publishers

Specialties Genetics
Pharmacology

Date 2021 Apr 27

PMID 33902406

Citations 30

Authors

Cynthia Amador

Ruchi Shah

Sean Ghiam

Andrei A Kramerov

Alexander V Ljubimov

Affiliations

Soon will be listed here.

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.

Citing Articles

Looking to the Future of Viral Vectors in Ocular Gene Therapy: Clinical Review.

Kharisova C, Kitaeva K, Solovyeva V, Sufianov A, Sufianova G, Akhmetshin R Biomedicines. 2025; 13(2).

PMID: 40002778 PMC: 11852528. DOI: 10.3390/biomedicines13020365.

Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions.

Banou L, Sarrafpour S, Teng C, Liu J Yale J Biol Med. 2024; 97(4):491-503.

PMID: 39703610 PMC: 11650918. DOI: 10.59249/HWID7537.

Gene Therapy: Towards a New Era of Medicine.

Bhagat M, Kamal R, Sharma J, Kaur K, Sharma A, Thakur G AAPS PharmSciTech. 2024; 26(1):17.

PMID: 39702810 DOI: 10.1208/s12249-024-03010-6.

Gene therapy for glaucoma: Targeting key mechanisms.

Henderson J, OCallaghan J, Campbell M Vision Res. 2024; 225:108502.

PMID: 39423611 PMC: 11579448. DOI: 10.1016/j.visres.2024.108502.

Sustained and Efficient Delivery of Antivascular Endothelial Growth Factor by the Adeno-associated Virus for the Treatment of Corneal Neovascularization: An Outlook for Its Clinical Translation.

Xie M, Wang L, Deng Y, Ma K, Yin H, Zhang X J Ophthalmol. 2024; 2024:5487973.

PMID: 39286553 PMC: 11405113. DOI: 10.1155/2024/5487973.

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