GeneticsThe CRISPR JournalPeer-reviewed
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…·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.
116 citationstop 2%