Adeno-associated virus packaging systems
AssigneeOxford Biomedica Solutions LLC
InventorsLaura VAN LIESHOUT, Marissa STANVICK
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Abstract
Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV). The dual vector transfection system generally comprises: (1) a first nucleic acid vector comprising a first nucleotide sequence encoding an AAV Rep protein, a second nucleotide sequence comprising an rAAV genome comprising a transgene, and a third nucleotide sequence encoding an AAV capsid protein; and (2) a second nucleic acid vector comprising a helper virus gene.
Claims30
A first nucleic acid vector comprising:
- a first nucleotide sequence encoding an AAV Rep protein;
- a second nucleotide sequence comprising a recombinant AAV (rAAV) genome comprising a transgene;
- and
- a third nucleotide sequence encoding an AAV capsid protein,
- wherein the nucleic acid vector does not comprise a helper virus gene.
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A recombinant AAV (rAAV) packaging system, comprising:
- (i) a first nucleic acid vector comprising:
- a first nucleotide sequence encoding an AAV Rep protein;
- a second nucleotide sequence comprising a recombinant AAV (rAAV) genome comprising a transgene;
- and
- a third nucleotide sequence encoding an AAV capsid protein, and
- (ii) a second nucleic acid vector comprising a helper virus gene.
7-15. (canceled)
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28-30. (canceled)
A recombinant packaging system comprising:
- (i) a first nucleic acid vector comprising:
- a first nucleotide sequence encoding an AAV Rep protein;
- a second nucleotide sequence comprising a recombinant AAV (rAAV) genome comprising a transgene;
- and
- a third nucleotide sequence encoding an AAV capsid protein, and
- (ii) a second nucleic acid vector comprising a nucleotide sequence that is at least 85% identical to a nucleotide sequence set forth in SEQ ID NO:
- 60, 61, 62, or 63.
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A host cell comprising:
- (i) a first nucleic acid vector comprising:
- a first nucleotide sequence encoding an AAV Rep protein;
- a second nucleotide sequence comprising a recombinant AAV (rAAV) genome comprising a transgene;
- and
- a third nucleotide sequence encoding an AAV capsid protein, and
- (ii) a second nucleic acid vector comprising a helper virus gene.
A method for recombinant preparation of an rAAV, the method comprising introducing the packaging system comprising:
- (i) a first nucleic acid vector comprising:
- a first nucleotide sequence encoding an AAV Rep protein;
- a second nucleotide sequence comprising a recombinant AAV (rAAV) genome comprising a transgene;
- and
- a third nucleotide sequence encoding an AAV capsid protein, and
- (ii) a second nucleic acid vector comprising a helper virus gene into a mammalian cell under conditions whereby the rAAV is produced.
43-50. (canceled)
54-56. (canceled)
Dependencies are inferred from the claim text.