Patent ID: 8236557

Claim:
A hybrid dual AAV (hdAAV) vector system comprising: (a) a first recombinant AAV vector comprising: (i) a first DNA segment comprising a 5′-inverted terminal repeat (5′-ITR) of AAV, (ii) a second DNA segment comprising a first portion of an open reading frame of a target gene operably linked to a promoter, (iii) a third DNA segment comprising a splice donor site, (iv) a fourth DNA segment comprising an alkaline phosphatase (AP) gene fragment, and (v) a fifth DNA segment comprising a 3′-inverted terminal repeat (3′-ITR) of AAV; and (b) a second recombinant AAV vector comprising: (i) a first DNA segment comprising a 5′-ITR of AAV, (ii) a second DNA segment comprising the AP gene fragment, (iii) a third DNA segment comprising a splice acceptor site, (iv) a fourth DNA segment comprising a second portion of the open reading frame of the target gene, which together with the first portion of the open reading frame encodes the target gene (polypeptide), operably linked with a polyadenylation (pA) signal, and (v) a fifth DNA segment comprising a 3′-ITR of AAV.