Adeno-associated virus (AAV) vector and uses therefor
AssigneeThe Trustees of the University of Pennsylvania
InventorQiang Wang
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Abstract
Provided herein are engineered nucleic acid sequences encoding AAVhu68 which provide improved yields of packaged AAVhu68 viral particles. Also provided is a packaging host cell comprising the engineered nucleotide sequence and methods of using same to product recombinant AAVhu68 vectors useful for gene delivery. Further provided are production cell supernatants comprising improved yields of recombinant AAV vectors having AAVhu68 capsids produced using the nucleotide sequences provided herein.
Claims18
A production host cell for generating a recombinant adeno-associated virus (rAAV) having an AAVhu68 capsid comprising:
- (a) a nucleic acid sequence of SEQ ID NO:
- 1 or a sequence at least 99% identical thereto encoding the AAVhu68 vp1 capsid protein operably linked to expression control sequences which direct expression of the AAVhu68 vp1 capsid protein in the host cell;
- (b) a nucleic acid molecule for packaging into the AAVhu68 capsid, said nucleic acid molecule comprising at least one AAV inverted terminal repeat (ITR) and a non-AAV nucleic acid sequence encoding a gene product operably linked to sequences which direct expression of the product in a host cell;
- and
- (c) sufficient AAV rep functions and helper functions to permit packaging of the nucleic acid molecule into the AAVhu68 capsid.
Dependencies are inferred from the claim text.