Allele-specific CRISPR/Cas9 genome editing of the single-base P23H mutation for rhodopsin associated dominant retinitis pigmentosa
Pingjuan Li, Benjamin P. Kleinstiver, Mihoko Leon, Michelle S. Prew, Daniel Navarro-Gomez, Scott H. Greenwald, Eric A. Pierce, J. Keith Joung, Qin Liu
This study demonstrates allele-specific CRISPR/Cas9 editing of the P23H mutation in Rho-P23H mice, reducing mutant allele expression and delaying photoreceptor degeneration.
The CRISPR Journal · Feb 1, 2018Peer-reviewedGenetics