Adeno-associated virus serotype i nucleic acid sequences, vectors and host cells containing same

Abstract

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 . (canceled) 
     
     
         2 . A composition comprising 1×10 9  to 1×10 16  genomes of a recombinant adenovirus having an AAV1 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, and a pharmaceutically acceptable carrier. 
     
     
         3 . The composition according to  claim 2 , comprising 1×10 13  to 1×10 16  genomes. 
     
     
         4 . The composition according to  claim 1 , wherein said vp1 protein has the amino acid sequence of SEQ ID NO: 13. 
     
     
         5 . The composition according to  claim 1 , wherein said vp2 protein has the amino acid sequence of SEQ ID NO: 15. 
     
     
         6 . The composition according to  claim 1 , wherein the 5′ ITR and 3′ ITR are of AAV serotype 2. 
     
     
         7 . The composition according to  claim 1 , further comprising a promoter which directs expression of the transgene. 
     
     
         8 . The composition according to  claim 1 , wherein said transgene encodes a protein or peptide. 
     
     
         9 . The composition according to  claim 8 , wherein said protein or peptide is a therapeutic protein or peptide. 
     
     
         10 . The composition according to  claim 8 , wherein said protein or peptide is an immunogenic protein or peptide. 
     
     
         11 . The composition according to  claim 1 , wherein said transgene encodes a cytokine, a hormone, or a growth factor. 
     
     
         12 . The composition according to  claim 1 , wherein said composition is formulated for delivery to muscle. 
     
     
         13 . The composition according to  claim 7 , wherein the promoter is a cytomegalovirus promoter.