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. Author manuscript; available in PMC: 2009 Dec 28.
Published in final edited form as: Gene Ther. 2009 Feb 19;16(5):581–588. doi: 10.1038/gt.2009.16

Figure 1.

Figure 1

Recombinant adeno-associated viral vectors (rAAV)-mediating gene-targeting approaches used to generate new cystic fibrosis ferret and pig models. (a) Schematic representation of the mechanism of gene targeting using rAAV. Target sequence homology (blue) and a selectable marker (red) are packaged between two AAV inverted terminal repeats (ITRs). Following infection, homologous recombination between the rAAV vector and the target genomic locus mediates insertion of the selectable marker. (b) Design of rAAV vectors used to disrupt the coding sequence of the CFTR gene through targeted insertion of a neomycin expression cassette in exon 10. (c) Design of rAAV vectors used to delete the F508 codon in exon 10 of the CFTR gene. In this approach, the neomycin expression cassette is inserted into intron 11 of the CFTR gene.