Update on Viral Gene Therapy Clinical Trials for Retinal Diseases.
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ABSTRACT:
SUBMITTER: Cheng SY
PROVIDER: S-EPMC9639220 | biostudies-literature | 2022 Sep
REPOSITORIES: biostudies-literature
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Update on Viral Gene Therapy Clinical Trials for Retinal Diseases.
Cheng Shun-Yun SY Punzo Claudio C
Human gene therapy 20220901 17-18
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 lea ...[more]
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