Skip to main content
. Author manuscript; available in PMC: 2014 Jul 15.
Published in final edited form as: Expert Opin Drug Deliv. 2014 Jan 3;11(3):345–364. doi: 10.1517/17425247.2014.871258

Figure 1. Schematics depicting four dual vector designs for delivering large genes using rAAV vector.

Figure 1

In all designs, two rAAV DNA genomes from separate vectors join together through either ITR-dependent or ITR-independent recombination. The recombined DNA is transcribed into pre-mRNA (not shown), which further undergoes splicing in trans-splicing and hybrid dual vector designs. The mature mRNA is translated into therapeutic proteins. See text for details.

Inline graphic: ITR; E: Enhancer; I: Intron; ITR: Inverted terminal repeat; P: Promoter; pA: Polyadenylation signal; rAAV: recombinant adeno-associated virus; SA: Splicing acceptor; SD: Splicing donor; Tg: Transgene.