Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
AssigneeThe Trustees of the University of Pennsylvania
InventorsGuangping Gao, James M. Wilson, Mauricio R. Alvira
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Abstract
Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. rAAV8-mediated delivery of therapeutic and immunogenic genes, including Factor VIII, is also provided.
Claims18
An isolated adeno-associated virus (AAV) comprising an AAV8 capsid having an amino acid sequence of amino acids 1 to 737 of SEQ ID NO:2 or a sequence which is at least 95% identical therewith.
An isolated capsid protein comprising an AAV8 protein selected from the group consisting of:
- vp1capsid protein having the sequence of amino acids (aa) 1 to 737 of SEQ ID NO:2;
- vp2 capsid protein having the sequence of aa 138 to 737 of SEQ ID NO:2;
- and
- vp3 capsid protein having the sequence of aa 203 to 737 of SEQ ID NO:2.
A recombinant adeno-associated viral (AAV) vector comprising an AAV8 capsid and a nucleotide sequence encoding a Factor VIII transgene operably linked to expression control elements.
A method of treating hemophilia in a mammal comprising:
- administering a rAAV having a serotype 8 capsid and a nucleic acid sequence encoding a Factor VIII transciene to a mammal under conditions that result in the expression of the Factor VIII protein at a level that provides a therapeutic effect in said mammal.
A viral vector comprising an AAV8 capsid and a transgene encoding a product under control of regulatory elements that direct expression of the product in a host cell.
An isolated AAV8 rep protein having the amino acid sequence of amino acids 1 to 625 of SEQ ID NO:3.
An isolated AAV8 rep protein fragment selected from the group consisting of amino acid (aa) 1 to 102;
- aa 103 to 140;
- aa 141 to 173;
- aa 174 to 226;
- aa 227 to 275;
- aa 276 to 374;
- aa 375 to 383;
- aa 384 to 446;
- aa 447 to 542;
- aa 543 to 555;
- and aa 556 to 625, of SEQ ID NO:
- 3.
Dependencies are inferred from the claim text.