Gene therapy vector based on adeno-associated virus type-2

Abstract

Objective: To construct an adeno-associated virus vector(AAV) plasmid used for gene therapy of central nervous system diseases. Methods: By using recombinant DNA techniques, a universal adeno-associated virus vector pssHG-Neo was constructed, which was derived from a wild type adeno-associated virus type 2 plasmid pssv9int- and a retrovirus plasmid plxsn. Results: This vector plasmid contained procaryotic cells propagating and selecting genes: PUC origin and ampicillin resistance (Ampr) gene. It also contained a multiple cloning sites(MCS) and an eucaryotic cells' selecting gene: neomycin resistance (Necr) gene. 5' LTR (long terminal repeat sequence) of retrovirus, which was located at the upstream of MCS, functioned as a promoter to control the foreign gene inserted in MCS. Since this plasmid flanked with AAV ITRs (inverted terminal repeat sequence), the foreign gene will be stably integrated into specific locus of human chromosome 19 (AAVS1). Conclusion: AAV vectors will be promising gene therapy vectors.