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

Benjamin Bakondi, Wenjian Lv, Bin Lu, Melissa K. Jones, Yuchun Tsai, Kevin J. Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J. Breunig, Clive N. Svendsen, Shaomei Wang

Research output: Contribution to journalArticlepeer-review

223 Scopus citations

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.

Original languageEnglish
Pages (from-to)556-563
Number of pages8
JournalMolecular Therapy
Volume24
Issue number3
DOIs
StatePublished - 1 Mar 2016
Externally publishedYes

Fingerprint

Dive into the research topics of 'In vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa'. Together they form a unique fingerprint.

Cite this