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Update on ocular gene therapy and advances in treatment of inherited retinal diseases and exudative macular degeneration

Garoon, Robert B.; Stout, J. Timothy

Current Opinion in Ophthalmology: May 2016 - Volume 27 - Issue 3 - p 268–273
doi: 10.1097/ICU.0000000000000256
TRANSLATIONAL RESEARCH: Edited by Jason Hsu and Sunir J. Garg

Purpose of review The purpose of this article is to provide an update on ocular gene therapy and discuss current active clinical trials.

Recent findings The main target for ocular gene therapy involves the retinal pigment epithelium or photoreceptors. The most common method to deliver viral vectors to these cells includes intravitreal injection, subretinal injection, or access from the suprachoroidal space. Recombinant adeno-associated virus and lentivirus can be engineered to maximize gene delivery to specific targets. There are several clinical trials currently aimed at treating inherited and retinal diseases with gene therapy via viral vectors.

Summary Recent advances in gene therapy have allowed for a better understanding of inherited and proliferative retinal diseases. New techniques have been developed to improve delivery of viral vectors to their cellular targets. There are currently multiple active clinical trials involving gene therapy underway with promising preliminary results.

aCullen Eye Institute

bSchool of Medicine, Baylor College of Medicine, Houston, Texas, USA

Correspondence to J. Timothy Stout, MD, PhD, MBA, Sid W. Richardson Professor, Margarett Root Brown Chair Director, Cullen Eye Institute, Baylor College of Medicine, 6565 Fannin NC205, Houston, TX 77030, USA. Tel: +1 713 798 5951; fax: +1 713 798 3026; e-mail:

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