Targeted In Situ Gene Correction of Dysfunctional APOE Alleles to Produce Atheroprotective Plasma ApoE3 Protein

Figure 4

Conversion of the mutant APOE2 allele to wild-type APOE3 using an AAV-based DNA repair sequence. The single-stranded adeno-associated virus genome, which contains Exon 4 of APOE3 and its flanking regions within its ITRs (inverted terminal repeats), is an efficient recombination template. As indicated, the mechanism is via a homologous recombination (HR) pathway.