CELL-LINES FOR THE PRODUCTION OF RECOMBINANT ADENOASSOCIATED VIRUS

Adeno-associated virus (AAV) is a replication-defective parvovirus tha t is being developed as a vector for human gene transfer, However, a m ajor obstacle to commonplace usage of AAV vectors is the production of recombinant virions (rAAV) in sufficient quantities for not only huma n trials, but also for preclinical studies of basic biology, toxicolog y, and efficacy, Unfortunately, current methods for large-scale produc tion are cumbersome and expensive, We have developed a simplified meth od for generating rAAV by establishing neomycin-resistant cell lines c ontaining copies of the AAV rep-cap genes and a rAAV vector, After inf ection with adenovirus, these cell lines were shown to produce infecti ous rAAV in relatively high titer, This method eliminates the need for exogenous DNA transfection and scale-up procedures are limited only b y the normal constraints of growing cells in culture.