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US 7,790,449 B2Granted
Adeno-associated virus (AAV) serotype 8 sequences, vectors containing the 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.
Claims14
An adeno-associated virus (AAV) 8 vector comprising a capsid comprising an AAV8 vp3 protein having the sequence of amino acid 203 to 737 of SEQ ID NO:
- 2 or a sequence at least 95% identical thereto, said vector further comprising a minigene comprising AAV inverted terminal repeats and a heterologous nucleic acid sequence operably linked to regulatory sequences which direct expression of a product from the heterologous nucleic acid sequence in a host cell.
Dependencies are inferred from the claim text.