Patent ID: 9150882
Filing Date: 2015-10-06
Classification: A61K,C12N

Abstract:
1. A parvovirus vector template nucleic acid for creating a self-complementary, genetically stable, infectious recombinant, adeno-associated virus (rAAV) virion comprising: a) a 5′ inverted terminal repeat (ITR); b) a 3′ ITR; and c) a foreign DNA insert comprising one or more expression cassettes, each independently ranging in size from about 0.2 kb to about 2.2 kb, and adding up to a total size of no more than 2.4 kb in length between the 5′ITR and the 3′ ITR; wherein (i) the 5′ and the 3′ ITR have between 50 and 80% complementarity to each other, (ii) the 3′ ITR serves as a primer for DNA replication, (iii) the 5′ or 3′ ITR is from AAV-2 and the other ITR is from AAV-4, and (iv) at least one of the 5′ or 3′ ITRs does not contain a functional terminal resolution site (trs).