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Update on Viral Gene Therapy Clinical Trials for Retinal Diseases

Cheng, Shun-Yun
Punzo, Claudio
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Journal Article
Publication Date
2022-09-16
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Abstract

In 2001, the first large animal was successfully treated with a gene therapy that restored its vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness called Leber's congenital amaurosis type 2. Sixteen years later, the gene therapy was approved by the U.S. Food and Drug Administration. The success of this gene therapy in dogs led to a fast expansion of the ocular gene therapy field. By now every class of inherited retinal dystrophy has been treated in at least one animal model and many clinical trials have been initiated in humans. In this study, we review the status of viral gene therapies for the retina, with a focus on ongoing human clinical trials. It is likely that in the next decade we will see several new viral gene therapies approved.

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Cheng SY, Punzo C. Update on Viral Gene Therapy Clinical Trials for Retinal Diseases. Hum Gene Ther. 2022 Sep;33(17-18):865-878. doi: 10.1089/hum.2022.159. PMID: 36074935; PMCID: PMC9639220.

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DOI
10.1089/hum.2022.159
PubMed ID
36074935
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Copyright Shun-Yun Cheng and Claudio Punzo 2022; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons License [CC-BY] (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.Attribution 4.0 International