METHODS AND VECTOR CONSTRUCTS USEFUL FOR PRODUCTION OF RECOMBINANT AAV

摘要

Methods for efficient production of recombinant AAV employ a host cell containing a first nucleic acid molecule comprising from 5' to 3', a parvovirus P5 promoter, a spacer, an AAV rep sequence and an AAV cap gene sequence, wherein said spacer is of sufficient size to reduce expression of the rep78 and rep68 gene products; a second nucleic acid molecule comprising a minigene comprising a transgene flanked by AAV inverse terminal repeats (ITRs) and under the control of regulatory sequences directing expression thereof in a host cell; and helper functions essential to the replication and packaging of rAAV, which functions are not provided by the first or second nucleic acid molecules. Host cells and molecule constructs are also described.