Fig. 3.

Allele-specific targeting of a pathogenic allele results in gene conversion. a Allele-specific sgRNA design strategy to target the CCDC40 allele 1 (Δ-2nt) in the PCD_02:30 hiPSC line. The protospacer sequence is underlined in red, and the protospacer adjacent motif in black. b Targeting of Δ-2nt allele by transducing PCD_02:30 cells with LF-CRISPR/Cas9-CCDC40-YGT particles at 0.5, 2, and 5 pg p24/cell results in dose-dependent interallelic gene conversion. Top left: Allelic composition at the targeted locus. Top right: Wild type (WT) allele copy number was measured by droplet digital PCR (ddPCR). Bottom: Representative Sanger sequencing chromatograms. ICE, Inference of CRISPR Edits; Hom, homozygote; NT, not transduced