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Mol Ther. 2016 Mar;24(3):556-63. doi: 10.1038/mt.2015.220. Epub 2015 Dec 15.

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

Author information

1
Board of Governors Regenerative Medicine Institute, Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, California, USA.
2
Current address: Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.

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 (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 of Rho(S334), which prevented retinal degeneration and improved visual function.

PMID:
26666451
PMCID:
PMC4786918
DOI:
10.1038/mt.2015.220
[Indexed for MEDLINE]
Free PMC Article

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