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10.1038/mt.2015.220

http://scihub22266oqcxt.onion/10.1038/mt.2015.220
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suck abstract from ncbi


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pmid26666451      Mol+Ther 2016 ; 24 (3): 556-63
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  • In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa #MMPMID26666451
  • Bakondi B; Lv W; Lu B; Jones MK; Tsai Y; Kim KJ; Levy R; Akhtar AA; Breunig JJ; Svendsen CN; Wang S
  • Mol Ther 2016[Mar]; 24 (3): 556-63 PMID26666451show ga
  • Reliable 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.
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