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Site-Specific Editing of CF Airway Basal Cells
(A) Site-specific correction of ΔF508 mediated by targeted nuclease cleavage with wild-type DNA template serving as donor. (B) Integration of CFTR9-27 cDNA (encoding exons 9–27), preceded by a splice acceptor (SA), followed by polyadenylation (pA) sequences, and flanked by homology sequences, into CFTR intron 8; the spliced mRNA joins endogenous exon 8 with the exon 9–27 transgene.
