IMR Press / FBE / Volume 4 / Issue 7 / DOI: 10.2741/E565

Frontiers in Bioscience-Elite (FBE) is published by IMR Press from Volume 13 Issue 2 (2021). Previous articles were published by another publisher on a subscription basis, and they are hosted by IMR Press on imrpress.com as a courtesy and upon agreement with Frontiers in Bioscience.

Open Access Review

Gene therapy in age related macular degeneration and hereditary macular disorders

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1 Department of Ophthalmology, University of Eastern Finland, P.O. Box 1627, FIN-70211 Kuopio, Finland
2 Department of Ophthalmology, Kuopio University Hospital, P.O. Box 1777, FIN-70211 Kuopio, Finland
3 Department of Biotechnology and Molecular Medicine, A.I.Virtanen Institute, University of Eastern Finland, P.O. Box 1627, FIN-70211 Kuopio, Finland
4 Department of Medicine, University of Eastern Finland, P.O. Box 1627, FIN-70211 Kuopio, Finland
5 Gene Therapy Unit, Kuopio University Hospital, P.O. Box 1777, FIN-70211Kuopio, Finland

*Author to whom correspondence should be addressed.

Academic Editor: Kai Kaarniranta

Front. Biosci. (Elite Ed) 2012, 4(7), 2546–2557; https://doi.org/10.2741/E565
Published: 1 June 2012
(This article belongs to the Special Issue Pathogenetic mechanism of age-related macular degeneration)
Abstract

In ophthalmology, administration of the therapeutic agent can be difficult due to the tight barriers in the eye. Multiple injections may be needed to allow the therapeutic agent to reach adequate levels in retina and choroidea which may increase the risk of complications including endophthalmitis, cataract and haemorrhages. Optimal methods for the delivery of therapeutic agents to the posterior segments of the eye have not yet been developed. Gene therapy offers an alternative where the therapeutic protein or proteins can be induced in the target tissue for a prolonged period of time after a single injection. The eye is a promising target for gene therapy due to its small size and tissue boundaries preventing leakage of the therapeutic material to other tissues or systemic circulation. However, most of the work in ocular gene therapy is still at the preclinical phase; only three vectors have reached phase 1/2 clinical trials. This review summarizes basic principles and current status of gene therapy in age related macular degeneration and hereditary macular disorders.

Keywords
Choroideal neovascularization
Gene therapy
Ocular hereditary disorders
Retinal neovascularization
Viral vectors
Non-viral vectors
Review
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