Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby

Patent metadata

Field	Value
Publication number	US-11034977-B2
Application number	US-201916698412-A
Country	US
Kind code	B2
Filing date	Nov 27, 2019
Priority date	Nov 13, 2001
Publication date	Jun 15, 2021
Grant date	Jun 15, 2021

Abstract

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

First claim

The invention claimed is:

1. A recombinant adeno-associated virus (AAV) comprising an AAV capsid comprising vp1 proteins having the sequence of amino acids 1 to 738 of SEQ ID NO: 85 or a sequence at least 95% identical to the full length of amino acids 1 to 738 of SEQ ID NO: 85, AAV vp2 proteins, and AAV vp3 proteins, wherein the recombinant AAV further comprises, packaged within the capsid, a 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 gene product in a host cell, wherein the gene product is a dystrophin protein.

2. The recombinant AAV according to claim 1 , wherein the sequence of the vp1 proteins is at least 97% identical to the full length of amino acids 1 to 738 of SEQ ID NO: 85.

3. The recombinant AAV according to claim 1 , wherein the sequence of the vp1 proteins is at least 99% identical to the full-length of amino acids 1 to 738 of SEQ ID NO: 85.

4. The recombinant AAV according to claim 1 , wherein the sequence of the vp1 proteins is the full-length of amino acids 1 to 738 of SEQ ID NO: 85.

5. A recombinant adeno-associated virus (AAV) having an AAV capsid comprising AAV vp1 proteins, AAV vp2 proteins having a sequence of amino acids 138 to 738 of SEQ ID NO:85 or a sequence at least 95% identical to the full-length of amino acids 138 to 738 of SEQ ID NO: 85, and AAV vp3 proteins, wherein the recombinant AAV further comprises, packaged within the capsid, a 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 gene product in a host cell, wherein the gene product is a dystrophin protein.

6. The recombinant AAV according to claim 5 , wherein the sequence of the vp2 proteins is at least 97% identical to the full length of amino acids 138 to 738 of SEQ ID NO: 85.

7. The recombinant AAV according to claim 5 , wherein the sequence of the vp2 proteins is at least 99% identical to the full-length of amino acids 138 to 738 of SEQ ID NO: 85.

8. The recombinant AAV according to claim 5 , wherein the sequence of the vp2 proteins is the full-length of amino acids 138 to 738 of SEQ ID NO: 85.

9. A recombinant adeno-associated virus (AAV) having an AAV capsid comprising AAV vp1 proteins, AAV vp2 proteins, and AAV vp3 proteins having a sequence of amino acids 204 to 738 of SEQ ID NO:85 or a sequence at least 95% identical to the full-length of amino acids 204 to 738 of SEQ ID NO: 85, wherein the recombinant AAV further comprises, packaged within the capsid, a 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 gene product in a host cell, wherein the gene product is a dystrophin protein.

10. The recombinant AAV according to claim 9 , wherein the sequence of the vp3 proteins is at least 97% identical to the full length of amino acids 204 to 738 of SEQ ID NO: 85.

11. The recombinant AAV according to claim 9 , wherein the sequence of the vp3 proteins is at least 99% identical to the full-length of amino acids 204 to 738 of SEQ ID NO: 85.

12. The recombinant AAV according to claim 9 , wherein the sequence of the vp3 proteins is the full-length of amino acids 204 to 738 of SEQ ID NO: 85.

13. The recombinant AAV according to claim 1 , wherein the AAV ITR is from AAV2.

14. The recombinant AAV according to claim 5 , wherein the AAV ITR is from AAV2.

15. The recombinant AAV according to claim 9 , wherein the AAV ITR is from AAV2.

16. A composition comprising a pharmaceutically compatible carrier and at least the recombinant AAV according to claim 1 .

17. A composition comprising a pharmaceutically compatible carrier and at least the recombinant AAV according to claim 5 .

18. A composition comprising a pharmaceutically compatible carrier and at least the recombinant AAV according to claim 9 .

19. A method for delivering a transgene product to a subject, said method comprising administering the recombinant AAV according to claim 1 .

20. A method for delivering a transgene product to a subject, said method comprising administering the recombinant AAV according to claim 5 .

21. A method for delivering a transgene product to a subject, said method comprising administering the recombinant AAV according to claim 9 .

Assignees

• Univ Pennsylvania

Inventors

• Gao Guangping
• Wilson James M
• Alvira Mauricio R

Classifications

• C07K14/005 Primary

  from viruses · CPC title

• C12Q1/701 Primary

  Specific hybridization probes · CPC title

• C12Q2600/158

  Expression markers · CPC title

• C12N2750/14151

  Methods of production or purification of viral material · CPC title

• A61P3/10

  for hyperglycaemia, e.g. antidiabetics · CPC title