Replication competent AAV helper functions
OwnerSikun Liinventor
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Abstract
The present invention provides methods and compositions for the production of high titer, wild-type-free recombinant AAV (“rAAV”) virions. The compositions of the present invention include novel nucleic acids encoding replication competent but packaging defective AAV helper functions and AAV helper function vectors. The present invention also includes host cells transfected by the claimed nucleic acids, methods of using the claimed vectors, and rAAV virions produced by such methods. The similar strategy can also be used for autonomous parvovirus virion production.
Claims34
An isolated nucleic acid molecule encoding an adeno-associated virus (AAV) helper function, said nucleic acid molecule comprising:
- at least one copy of an AAV inverted terminal repeat (ITR) sequence or a functional equivalent thereof that is capable of supporting AAV replication;
- an AAV rep coding region;
- and
- an AAV cap coding region.
An isolated nucleic acid molecule encoding an autonomous parvovirus helper function, said nucleic acid molecule comprising:
- at least one copy of a parvovirus inverted terminal repeat (ITR) sequence or a functional equivalent thereof that is capable of supporting the autonomous parvovirus replication;
- an autonomous parvovirus non-structural protein coding region;
- and
- an autonomous parvovirus cap protein coding region.
Dependencies are inferred from the claim text.