In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

Reliable genome editingvia Clustered Regularly Interspaced Short PalindromicRepeat ( CRISPR)/ Cas9may provide a means to correct inherited diseasesin patients. As proof of principle, we show that CRISPR/ Cas9can be used in vivo to selectively ablate the rhodopsingene carrying the dominant S334ter mutation (RhoS334) in rats that model severe autosomal dominant retinitis pigmentosa. A single subretinal injection of guide RNA/ Cas9plasmid in combination with electroporationgenerated allele-specific disruption of RhoS334, which prevented retinal degenerationand improved visual function.