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US 6,670,176 B1Granted
Adeno-associated virus capable of expressing factor IX protein and cells comprising the same
AssigneesNational Institutes of Health, University of Pittsburgh
InventorsRichard Jude Samulski, Christopher E. Walsh, Arthur W. Nienhuis, Johnson M. Liu, Jeffrey L. Miller
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Abstract
The subject invention concerns a recombinant adeno-associated virus vector characterized as being capable of delivering and expressing at least one mammalian gene into a genome of a mammalian host cell such that the expression of the gene is regulated in a tissue specific manner by cis-acting regulatory and promoter elements of the gene. A method of using this recombinant adeno-associated virus vector for therapeutic purposes is also provided.
Claims8
A recombinant adeno-associated virus vector comprising:
- (a) at least a portion of the adeno-associated virus genome;
- and
- (b) a nucleic acid sequence encoding a mammalian Factor IX protein, wherein the expression of the mammalian Factor IX protein is regulated by a liver specific cis-acting regulatory element heterologous to the nucleic acid sequence encoding the mammalian Factor IX protein.
Dependencies are inferred from the claim text.