Bakondi, BenjaminLv, WenjianLu, BinJones, Melissa KTsai, YuchunKim, Kevin JLevy, RachelleAkhtar, Aslam AbbasiBreunig, Joshua JSvendsen, Clive NWang, Shaomei2016-04-012016Bakondi, B., W. Lv, B. Lu, M. K. Jones, Y. Tsai, K. J. Kim, R. Levy, et al. 2016. “In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.” Molecular Therapy 24 (3): 556-563. doi:10.1038/mt.2015.220. http://dx.doi.org/10.1038/mt.2015.220.1525-0016http://nrs.harvard.edu/urn-3:HUL.InstRepos:26318752Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter mutation (RhoS334) in rats that model severe autosomal dominant retinitis pigmentosa. A single subretinal injection of guide RNA/Cas9 plasmid in combination with electroporation generated allele-specific disruption of RhoS334, which prevented retinal degeneration and improved visual function.en-USJournal Article2016-04-0110.1038/mt.2015.220