Based on several previous publications (Khan et al., 2010, 2011), we explored the utility of adeno-associated virus (AAV) as a method to improve gene targeting efficiencies in hPSCs. For SOX2, a gene that is highly expressed in undifferentiated hPSCs, gene targeting rates were greater than 70%. Similar targeting efficiencies in hPSCs using AAV have been reported by others (Asuri et al., 2012; Khan et al., 2010, 2011; Smith-Arica et al., 2003), indicating that AAV offers a highly efficient and robust approach to target genes for HR in hPSCs.
SOX2
gene targeting rates
greater than 70%
%