Increased prime edit rates in KCNQ2 and SCN1A via single nicking all-in-one plasmids.
N DirkxWout J WeuringE De VriendtN SmalJ van de VondervoortRuben van 't SlotM KoetsierN ZonnekeinTim De PooterS WeckhuysenB P C KoelemanPublished in: BMC biology (2023)
Taken together, our study shows an improved efficacy of EF-1α driven sncRNA pAIO-PE plasmids in hiPSC reaching high editing rates, especially after FACS sorting. Optimizing these sncRNA PE systems is of high value when considering future therapeutic in vivo use, where accuracy will be extremely important.