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Gene Therapy for Ocular Diseases 

Gene Therapy for Ocular Diseases
Gene Therapy for Ocular Diseases

Ben J. Kim

and Nadia K. Waheed

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date: 04 July 2022

Vision scientists are uniquely positioned to make contributions to the field of gene therapy. Because of its anatomy the eye provides several advantages over other organs as a site for gene therapy. Compared to other organs, the eye is both small and compartmentalized, and thus the number of cells requiring a delivered gene product is less than that of other organs. The relatively small amount of gene vector (such as a virus) required to affect ocular tissue, as well as the relatively tight blood-ocular barrier, limits potential complications from systemic exposure. A gene vector can also be delivered to a specific ocular tissue with direct visualization, whether it is subretinal, intravitreal, or within the anterior chamber (Fig. 52.1). Equally important, the eye is an “immune-privileged” organ, and this potentially limits the immune reaction to a given gene vector. In addition to these benefits regarding the delivery of gene therapy, the eye offers other advantages to enable one to study the effects of the gene therapy. The ocular tissues can be visualized, retinal function can be quantitated with electroretinography (ERG), and the fellow eye serves as a potential experimental control.1 Herein we will discuss current gene therapy modalities, including viral vectors, RNA interference, electrotransfer, and nanoparticles. Major gene therapy studies will be discussed with a focus on research for retinal diseases that demonstrates important concepts and has led to human clinical trials.

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