Novel aav capsids and compositions containing same
AssigneeThe Trustees of the University of Pennsylvania
InventorsKalyani Nambiar, James M. Wilson, Qiang Wang
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Abstract
Provided herein are novel AAV capsids and rAAV comprising the same. In one embodiment, vectors employing a novel AAV capsid show increased transduction of a selected target tissue as compared to a prior art AAV.
Claims15
1-9. (canceled)
A recombinant adeno-associated virus (rAAV) production system useful for producing an rAAV, wherein the production system comprises:
- (a) a nucleic acid sequence encoding the amino acid sequence of SEQ ID NO:
- 2;
- (b) a nucleic acid molecule suitable for packaging into an AAV 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 rAAV capsid.
A method of generating a rAAV comprising the steps of culturing a host cell in culture containing:
- (a) a nucleic acid molecule encoding an AAV capsid protein comprising the amino acid sequence of SEQ ID NO:
- 2;
- (b) a functional rep gene;
- (c) a vector genome comprising a AAV 5′ ITR, a AAV 3′ ITR, and a transgene;
- and (d) sufficient helper functions to permit packaging of the vector genome into an AAV capsid.
(canceled)
A method of delivering a transgene to a cell, said method comprising the step of contacting the cell with an rAAV having a capsid comprising capsid proteins produced by expression of the AAV capsid sequence of SEQ ID NO:
- 1 or 3, or a sequence sharing at least 90% identity with SEQ ID NO:
- 1 or 3, wherein said rAAV comprises the transgene.
A plasmid comprising a nucleic acid sequence encoding an AAV capsid protein, the nucleic acid sequence comprising at least SEQ ID NO:
- 1 or 3, or a sequence at least 70% identical to SEQ ID NO:
- 1 or 3 which encodes the amino acid sequence of SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.