Figure 1.

ZFN-Mediated Correction of ΔI507 or ΔF508 CFTR Mutations in CF iPSCs

(A) Outline of methodology involving co-delivery of CFTR-specific ZFNs together with CFTR donor, followed by Cre-recombinase-mediated excision.

(B) The schematic shows the expected genomic organization of a targeted CFTR allele including the WT exon 10 (shown in black) together with the pgk-puroTK selection cassette. A unique 6.4-kb hybridizing band is expected for a correctly modified clone and is apparent in the four corrected clones (17-14, 17-1, 17-16, and 17-9), but absent in the Cre-excised clones and the non-targeted clone 17 CF iPSCs.

(C) Sequence chromatograms of the modified WT and unmodified ΔF508 CFTR alleles from corrected CF iPSC clones.

See also Figure S1.