A genome-wide in vivo CRISPR screen identifies neuroprotective strategies in the mouse and human retina.

Abstract excerpt

Retinitis pigmentosa (RP) is a genetically diverse blinding disorder lacking broadly effective therapies. We performed a genome-wide in vivo CRISPR knockout screen in mice carrying the P23H rhodopsin mutation, the most common cause of autosomal dominant RP in the United States, to systematically identify neuroprotective genes. We discovered multiple knockouts that accelerated rod photoreceptor loss, validated top...

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