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 method for delivering a transgene to a host cell comprising delivery of a recombinant virus to a mammalian subject, wherein said recombinant virus has 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), said transgene, and an AAV 3′ ITR.
2. The method according to claim 1 , wherein the 5′ ITR and 3′ ITR of said recombinant virus are of AAV serotype 2.
3. The method according to claim 1 , wherein the 5′ ITR and 3′ ITR of said recombinant virus are of AAV serotype 1.
4. The method according to claim 1 , wherein said recombinant virus further comprises a promoter which directs expression of the transgene.
5. The method according to claim 4 , wherein said promoter is the cytomegalovirus (CMV) promoter.
6. The method according to claim 1 , wherein said transgene encodes a protein or peptide.
7. The method according to claim 6 , wherein said protein or peptide is a therapeutic protein or peptide.
8. The method according to claim 6 , wherein said protein or peptide is an immunogenic protein or peptide.
9. The method according to claim 1 , wherein said transgene encodes a cytokine, a hormone, or a growth factor.
10. The method according to claim 1 , wherein said transgene is alpha 1 anti-trypsin (α1AT).
11. The method according to claim 1 , wherein said transgene is erythropoietin (epo).
12. The method according to claim 1 , wherein said recombinant virus is formulated with a pharmaceutically acceptable carrier.
13. The method according to claim 1 , wherein said recombinant virus is delivered to muscle.
14. The method according to claim 1 , wherein said recombinant virus is delivered to liver.
15. The method according to claim 1 , wherein said recombinant virus is delivered intranasally.
16. The method according to claim 1 , wherein said vp1 protein has the amino acid sequence of SEQ ID NO: 13.
17. The method according to claim 1 , wherein said vp2 protein has the amino acid sequence of SEQ ID NO: 15.
18. The method according to claim 1 , wherein said recombinant virus is delivered intramuscularly.
19. The method according to claim 18 , wherein about 1×10 13 to 1×10 16 AAV genomes are delivered to said mammalian subject.
20. The method according to claim 1 , wherein said recombinant virus is delivered intravenously.