Article

Start codon disruption with CRISPR/Cas9 prevents murine Fuchs’ endothelial corneal dystrophy

2020-03-20

Abstract excerpt

A missense mutation of collagen type VIII alpha 2 chain (COL8A2) gene leads to early onset Fuchs’ endothelial corneal dystrophy (FECD), which progressively impairs vision through loss of corneal endothelial cells. We demonstrate that CRISPR/Cas9-based postnatal gene editing achieves structural and functional rescue in a mouse model of FECD. A single intraocular injection of an adenovirus encoding both the Cas9 gen...

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Literature Corpus work: 393218c7-eaa1-5a61-9e24-5a8d860d93a7
DOI: 10.1101/2020.03.18.996728

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