[HTML][HTML] In vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa

B Bakondi, W Lv, B Lu, MK Jones, Y Tsai, KJ Kim… - Molecular Therapy, 2016 - cell.com
B Bakondi, W Lv, B Lu, MK Jones, Y Tsai, KJ Kim, R Levy, AA Akhtar, JJ Breunig
Molecular Therapy, 2016cell.com
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 (Rho S334) 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 …
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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