Adeno-associated virus serotype I nucleic acid sequences, vectors and host cells containing same
View Patent ↗The nucleic acid sequences of adeno-associated virus (AAV) serotype 1 are provided, as are vectors and host cells containing these sequences and functional fragments thereof. Also provided are methods of delivering genes via AAV-1 derived vectors.
1. A recombinant virus having an AAV-1 capsid comprising a vp1 protein, a vp2 protein, and a vp3 protein, wherein said vp3 protein has an amino acid sequence of SEQ ID NO:17, wherein said recombinant virus further comprises a heterologous molecule which comprises an AAV 5′ inverted terminal repeat sequence (ITR), a transgene, and an AAV 3′ ITR.
2. The recombinant virus according to claim 1 , wherein said vp1 protein has the amino acid sequence of SEQ ID NO: 13.
3. The recombinant virus according to claim 1 , wherein said vp2 protein has the amino acid sequence of SEQ ID NO: 15.
4. The recombinant virus according to claim 1 , wherein the 5′ ITR and 3′ ITR are of AAV serotype 2.
5. The recombinant virus according to claim 1 , wherein the 5′ ITR and 3′ ITR are of AAV serotype 1.
6. The recombinant virus according to claim 1 , further comprising a promoter which directs expression of the transgene.
7. The recombinant virus according to claim 1 , wherein said transgene encodes a protein or peptide.
8. The recombinant virus according to claim 7 , wherein said protein or peptide is a therapeutic protein or peptide.
9. The recombinant virus according to claim 7 , wherein said protein or peptide is an immunogenic protein or peptide.
10. The recombinant virus according to claim 1 , wherein said transgene encodes a cytokine, a hormone, or a growth factor.
11. The recombinant virus according to claim 1 , wherein said transgene is alpha 1 anti-trypsin (α1AT).
12. The recombinant virus according to claim 1 , wherein said transgene is erythropoietin (epo).
13. An isolated host cell transformed with the recombinant virus of claim 1 .
14. A composition comprising a recombinant virus according to claim 1 and a pharmaceutically acceptable carrier.
15. The composition according to claim 14 , wherein said composition is formulated for delivery to muscle.
16. The composition according to claim 14 , wherein said composition is formulated for delivery to liver.
17. The composition according to claim 14 , wherein said composition is formulated for intranasal delivery.