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• W2761315124 abstract "No effective treatment exists for dominant inherited diseases. Here, we present a CRISPR/Cas9 genome editing strategy that combines multiple innovations to achieve specific and efficient disruption of the pathogenic allele with a single-nucleotide mutation (Rho-P23H) in a mouse model of dominant retinitis pigmentosa. Our study demonstrates the possibility of a spacer-mediated allele-specific editing approach, which may be applicable to a broad range of dominant disorders in which the mutant allele is not discriminable by placement of a PAM sequence." @default.
• W2761315124 created "2017-10-20" @default.
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• W2761315124 date "2017-10-03" @default.
• W2761315124 modified "2023-09-26" @default.
• W2761315124 title "Allele-specific editing of rhodopsin P23H knock-in mice broadens therapeutic potential of CRISPR/Cas for dominant genetic diseases" @default.
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• W2761315124 hasPublicationYear "2017" @default.
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