Granted Patent B2

US 8,962,332 · App. 14/025,951 · Granted Feb 24, 2015

Inventors: Guangping Gao (Westborough, MA); James M. Wilson (Glen Mills, PA); Mauricio R. Alvira (Philadelphia, PA)

Assignee: The Trustees of the University of Pennsylvania

A61K35/761C07K14/005C07K14/755C12N7/00C12N15/86A61K48/00C12N2750/14122C12N2750/14143C12N2830/008C12N2830/48C12N2830/85

Abstract

Claims (13)

1. A composition comprising a non-naturally occurring recombinant adeno-associated virus (AAV) comprising an AAV8 capsid comprising a vp1, vp2, and a vp3 protein, wherein said vp1 protein has an amino acid sequence which is at least about 95% identical to amino acids 1 to 737 of SEQ ID NO: 2, said composition further comprising a pharmaceutically acceptable carrier.

2. The composition according to claim 1 , wherein said vp1 protein has an amino acid sequence about 95% to 99% identical to amino acids 1 to 737 of SEQ ID NO: 2.

3. The composition according to claim 1 , wherein said vp1 protein has an amino acid sequence about 95% identical to amino acids 1 to 737 of SEQ ID NO: 2.

4. The composition according to claim 1 , wherein said vp1 protein has an amino acid sequence of amino acids 1 to 737 of SEQ ID NO: 2.

5. The composition according to claim 1 , further comprising a heterologous gene packaged therein, wherein said heterologous gene is operably linked to regulatory sequences which direct its expression.

6. The composition according to claim 5 , wherein said heterologous gene is flanked by one or more inverted terminal repeat (ITR) sequence from an AAV heterologous to AAV8.

7. The composition according to claim 6 , wherein the one or more AAV ITR is from AAV2.

8. A composition according to claim 1 , further comprising a preservative or chemical stabilizer.

9. A recombinant adeno-associated virus (AAV) comprising an AAV8 capsid comprising a vp1, vp2, and a vp3 protein, wherein said vp1 protein has an amino acid sequence at least about 95% identical with amino acids 1 to 737 of SEQ ID NO: 2, wherein said AAV further comprises a minigene having AAV inverted terminal repeat (ITR) sequences and a heterologous gene operably linked to regulatory sequences which direct its expression in a host cell.

10. The recombinant AAV according to claim 9 , wherein said vp1 protein has an amino acid sequence about 95% to 99% identical to amino acids 1 to 737 of SEQ ID NO: 2.

11. The recombinant AAV according to claim 9 , wherein said vp1 protein has an amino acid sequence about 95% identical to amino acids 1 to 737 of SEQ ID NO: 2.

12. The recombinant AAV according to claim 9 , wherein said vp1 protein has an amino acid sequence of amino acids 1 to 737 of SEQ ID NO: 2.

13. A composition comprising a recombinant AAV according to claim 9 and a pharmaceutically acceptable carrier.

Assignments (1)

Continuity (8)