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Department of Ophthalmology and Visual Sciences

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Congenital, Hereditary, and Neonatal Diseases and Abnormalities | Eye Diseases | Genetics and Genomics | Molecular, Cellular, and Tissue Engineering | Ophthalmology | Therapeutics


The eye is at the forefront of developing therapies for genetic diseases. With the FDA approval of the first gene-therapy drug for a form of congenital blindness, numerous studies have been initiated to develop gene therapies for other forms of eye diseases. These examinations have revealed new information about the benefits as well as restrictions to using drug-delivery routes to the different parts of the eye. In this article, we will discuss a brief history of gene therapy and its importance to the eye and ocular delivery landscape that is currently being investigated, and provide insights into their advantages and disadvantages. Efficient delivery routes and vehicle are crucial for an effective, safe, and longer-lasting therapy.


eye, gene therapy, retina

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Copyright © 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (

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Sahu B, Chug I, Khanna H. The Ocular Gene Delivery Landscape. Biomolecules. 2021 Aug 1;11(8):1135. doi: 10.3390/biom11081135. PMID: 34439800; PMCID: PMC8394578. Link to article on publisher's site

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Creative Commons Attribution 4.0 License
This work is licensed under a Creative Commons Attribution 4.0 License.