Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Guangping Gao, Mauricio R. Alvira, Luc H. Vandenberghe
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Abstract
Sequences of novel adeno-associated virus capsids 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. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
Claims7
A non-naturally occurring adeno-associated virus (AAV) comprising an AAV9 capsid wherein the AAV9 capsid comprises AAV9 capsid proteins with an amino acid sequence selected from the group consisting of:
- the amino acids (aa) 1 to 736 of SEQ ID NO:123;
- the aa 138 to 736 of SEQ ID NO:
- 123;
- and the aa 2O3 to 736 of SEQ ID NO:
- 123, said AAV further comprising a minigene having AAV inverted terminal repeats and a transgene comprising a heterologous gene operably linked to regulatory sequences which direct expression of the heterologous gene in a host cell.
An AAV comprising an AAV9 capsid, wherein the AAV9 capsid comprises an amino acid sequence that is at least 95% identical to amino acids 203 to 736 of SEQ ID NO:
- 123,and wherein said AAV further comprises a minigene having AAV inverted terminal repeats and a transgene comprising a heterologous gene operably linked to regulatory sequences which direct expression of the heterologous gene in a host cell, wherein said amino acid sequence is selected from the group consisting of SEQ ID NO:
- 121 and SEQ ID NO:
- 122.
Dependencies are inferred from the claim text.