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

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Author
Bakondi, Benjamin
Lv, Wenjian
Lu, Bin
Jones, Melissa K
Tsai, Yuchun
Kim, Kevin J
Levy, Rachelle
Akhtar, Aslam Abbasi
Breunig, Joshua J
Svendsen, Clive N
Wang, Shaomei
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https://doi.org/10.1038/mt.2015.220Metadata
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Bakondi, 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.Abstract
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.Other Sources
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786918/pdf/Terms of Use
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