Abstract
Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one early-onset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future.
Keywords:
clinical trials; gene therapy; retina.
Copyright © 2017 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.
Publication types
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Review
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
MeSH terms
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Adenoviruses, Human / genetics*
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Adenoviruses, Human / metabolism
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Animals
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Clinical Trials as Topic
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Disease Models, Animal
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Drug Approval
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Eye Proteins / genetics*
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Eye Proteins / metabolism
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Gene Expression
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Genetic Therapy / methods
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Genetic Therapy / trends*
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Genetic Vectors / administration & dosage*
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Genetic Vectors / chemistry
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Genetic Vectors / metabolism
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Humans
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Injections, Intraocular
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Mutation
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Retina / metabolism
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Retina / pathology
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Retinal Diseases / genetics
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Retinal Diseases / metabolism
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Retinal Diseases / pathology
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Retinal Diseases / therapy*