Claims
- 1. An isolated AAV-1 nucleic acid molecule comprising a sequence selected from the group consisting of:
(a) SEQ ID NO: 1; (b) a DNA sequence complementary to SEQ ID NO: 1; (c) cDNA complementary to (a) or (b); and (d) RNA complementary to any of (a) to (c).
- 2. A nucleic acid molecule comprising an AAV-1 inverted terminal repeat (ITR) sequence selected from the group consisting of:
(a) nt 1 to 143 of SEQ ID NO: 1; (b) nt 4576 to 4718 of SEQ ID NO: 1; (c) a nucleic acid sequence complementary to (a) or (b); and (d) a functional fragment of (a), (b), or (c).
- 3. A recombinant vector comprising a 5′ AAV-1 inverted terminal repeat (ITR) and a selected transgene, wherein said ITR has the sequence selected from the group consisting of:
(a) nt 1 to 143 of SEQ ID NO: 1; (b) a nucleic acid sequence complementary to (a); and (c) a functional fragment of (a) or (b).
- 4. The recombinant vector according to claim 3, wherein said vector further comprises a 3′ AAV-1 ITR.
- 5. A recombinant vector comprising a 3′ AAV-1 inverted terminal repeat (ITR) and a selected transgene, wherein said ITR has the sequence selected from the group consisting of:
(a) nt 4576 to 4718 of SEQ ID NO: 1; (b) a nucleic acid sequence complementary to (a); and (c) a functional fragment of (a) or (b).
- 6. The recombinant vector according to claim 5, wherein said vector further comprises a 5′ AAV-1 ITR.
- 7. A pharmaceutical composition comprising a carrier and a virus comprising the vector according to claim 5.
- 8. A method for producing a selected gene product comprising the steps of transfecting a mammalian cell with the molecule according to claim 1 or a functional fragment thereof and culturing said cell under conditions suitable to express said gene product.
- 9. The recombinant vector according to claim 3, wherein said vector further comprises AAV-1 capsid proteins having the sequence of SEQ ID NO: 13, 15 or 17 or functional fragments thereof.
- 10. The recombinant vector according to claim 3, wherein said vector further comprises adenovirus sequences.
- 11. The host cell transduced with a recombinant viral vector according to claim 3.
- 12. The host cell transduced with a nucleic acid molecule according to claim 1.
- 13. The host cell transduced with a nucleic acid molecule according to claim 2.
- 14. The pharmaceutical composition comprising a carrier and a virus comprising the vector according to claim 3.
- 15. The method for delivery of a transgene comprising the step of delivering to a host cell a recombinant virus comprising a recombinant vector according to claim 3.
- 16. A recombinant host cell transformed with a nucleic acid sequence expressing one or more AAV-1 rep proteins selected from among rep78 having the amino acid sequence of SEQ ID NO: 7, rep 68 having the amino acid sequence of SEQ ID NO: 7, rep 52 having the amino acid sequence of SEQ ID NO: 9, and rep 40 having the amino acid sequence of SEQ ID NO: 11.
- 17. A composition comprising a recombinant virus having an AAV-1 capsid comprising an AAV-1 protein selected from among AAV-1 vp1 having the amino acid sequence of SEQ ID No: 13; AAV-1 vp2 having the amino acid sequence of SEQ ID NO: 15 and AAV-1 vp3 having the amino acid sequence of SEQ ID NO: 17 and a heterologous molecule which comprises an AAV 5′ inverted terminal repeat sequence (ITR), a transgene, and an AAV 3′ ITR.
- 18. The composition of claim 17 wherein the AAV-1 protein vp1 is encoded by a nucleic acid having at least about 98% identity to nucleotides 2223-4431 of SEQ ID NO: 1, as measured by MacVector 6.0.
- 19. The composition of claim 17 wherein the AAV-1 protein vp2 is encoded by a nucleic acid having at least about 98% identity to nucleotides 2634-4432 of SEQ ID NO: 1, as measured by MacVector 6.0.
- 20. The composition of claim 17 wherein the AAV-1 protein vp3 is encoded by a nucleic acid having at least about 98% identity to nucleotides 2829-4432 of SEQ ID NO: 1, as measured by MacVector6.0.
- 21. The composition of claim 17 wherein the AAV 5′ ITR and 3′ ITR are of AAV serotype 2.
- 22. The composition of claim 21 wherein the recombinant virus further comprises a regulatable promoter which directs expression of the transgene.
CROSS-REFERENCE TO RELATED APPLICATIONS
[0001] This is a continuation of U.S. patent application Ser. No. 09/807,802, filed Nov. 29, 2001, which is a national phase of PCT/US99/25694, filed Nov. 2, 1999, which claims the benefit of the priority of U.S. patent application Ser. No. 60/107,114, filed Nov. 5, 1998.
STATEMENT REGARDING FEDERALLY SPONSORED RESEARCH OR DEVELOPMENT
[0002] This work was supported by the National Institutes of Health, grant no. P30 DK47757-06 and PO HD32649-04. The US government may have certain rights in this invention.
Provisional Applications (1)
|
Number |
Date |
Country |
|
60107114 |
Nov 1998 |
US |
Continuations (1)
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Number |
Date |
Country |
Parent |
09807802 |
Nov 2001 |
US |
Child |
10696900 |
Oct 2003 |
US |