Targeted integration of transfected and infected adeno-associated virus vectors containing the neomycin resistance gene.

Human adeno-associated virus (AAV) integrates specifically into a site (AAVS1) on chromosome 19q13.3-qter. We report that both transfected and infected AAV-vectors will target a foreign gene preferentially to AAVS1. Of 11 infected cell lines studied, nine (82%) showed integration of the neomycin resistance marker into AAVS1, which confirms the findings of previous investigations. We also show site-specific integration (nine out of 12 cell lines, 75%) following transfection of an AAV vector containing the neomycin gene. The ability of transfected AAV sequences to preferentially target and express selectable genes at a known site in the human genome extends the potential use of AAV as a vector in gene therapy.