Article

Limited time window for retinal gene therapy in a preclinical model of ciliopathy

2020-05-06

Abstract excerpt

<h4>ABSTRACT</h4> Retinal degeneration is a common clinical feature of ciliopathies, a group of genetic diseases linked to ciliary dysfunction, and gene therapy is an attractive treatment option to prevent vision loss. Although the efficacy of retinal gene therapy is well established by multiple proof-of-concept preclinical studies, its long-term effect, particularly when treatments are given at advanced disease...

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Literature Corpus work: 0ab5dccb-7020-5dd0-88eb-e71d6b6676c6
DOI: 10.1101/2020.05.05.072793

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