Table 1.

AAV virus-mediated marker transfer to patients

Vector platform	pUC plasmid (>2 kb pUC origin-antibiotic marker spacer region)	Minicircle (MC) retrofit (<100 bp spacer region)	Result-performance	Result-antibiotic marker gene transfer
Self-complementary AAV	pAAV-scGFP	MC.AAVscGFP	MC up to 30-fold improved transducing units27,a	plasmid backbone antibiotic resistance marker packaged in up to 26.1% viral particles27 potential for genome integration in transduced cells
Single-stranded AAV	AAV2-ssGFP	MC.AAVssGFP	MC up to 3-fold improved transducing units27,a	plasmid backbone antibiotic resistance marker packaged in up to 2.9%27 or 3%29 of viral particles potential for genome integration in transduced cells

^aNo improvement in viral titer compared with plasmid was observed with linear Doggybone DNA single-stranded AAV vector retrofits.³⁰ This suggests that short backbone minicircle vector improvement compared with >2 kb pUC-antibiotic marker plasmid backbone vectors require a short backbone circular vector rather than a linear vector such as Doggybone vectors.