Article

Gene editing preserves visual function in a mouse model of retinal degeneration

2019-05-02

Abstract excerpt

Inherited retinal dystrophies are a large and heterogeneous group of degenerative diseases caused by mutations in various genes. Given the favourable anatomical and immunological characteristics of the eye, gene therapy holds great potential for their treatment. We used a tailored CRISPR/Cas9-based gene editing system to prevent retinal photoreceptor death in the Rd10 mouse model of retinitis pigmentosa. We tested...

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Literature Corpus work: b44435a8-408c-5592-9469-5bac46ea9118
DOI: 10.1101/624858

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