Adenoviral vectors for enhanced gene expression
AssigneeGenetic Theraphy, Inc.
InventorsSheila Connelly, Michael Kaleko, Theodore Smith
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Abstract
An adenoviral vector including at least one DNA sequence encoding a clotting factor, such as, for example, Factor VIII, or Factor IX. Such vectors may be administered to a host in an amount effective to treat hemophilia in the host. The vectors infect hepatocytes very efficiently, whereby the hepatocytes express the DNA sequence encoding the clotting factor.
Claims106
An adenoviral vector comprising at least one heterologous DNA sequence and at least one intron which is not part of the adenoviral genome, wherein said vector is free of adenoviral DNA sequences encoding E1 proteins.
An adenoviral vector comprising at least one heterologous DNA sequence and at least one intron which is not part of the adenoviral genome, wherein said vector is free of at least the majority of adenoviral E1 and E3 DNA sequences.
An adenoviral vector comprising at least one heterologous DNA sequence and at least one intron selected from the group consisting of at least a portion of an intron of the Factor IX gene, the full seventh intron of the Factor IX gene, at least a portion of an intron of the Factor VIII gene and the first intron of the apolipoprotein A-1 gene, said at least one intron enhancing expression of said at least one heterologous DNA sequence.
A method of expressing a heterologous DNA sequence contained in an adenoviral vector, comprising operatively linking said heterologous DNA sequence with at least one intron selected from the group consisting of at least a portion of an intron of the Factor IX gene, the full seventh intron of the Factor IX gene, at least a portion of an intron of the factor VIII gene and the first intron of the apolipoprotein A-1 gene, said at least one intron enhancing expression of said heterologous DNA sequence.
An adenoviral vector comprising at least one heterologous DNA sequence, wherein said vector is free of adenoviral DNA sequences encoding E1 proteins and said at least one heterologous DNA sequence comprises at least one intron.
An adenoviral vector comprising at least one heterologous DNA sequence wherein said vector is free of at least the majority of adenoviral E1 and E3 DNA sequences and said at least one heterologous DNA sequence comprises at least one intron.
An adenoviral vector comprising at least one heterologous DNA sequence, said at least one heterologous DNA sequence comprising at least one intron selected from the group consisting of at least a portion of an intron of the Factor IX gene, the full seventh intron of the Factor IX gene, at least a portion of an intron of the Factor VIII gene and the first intron of the apolipoprotein A-1 gene, wherein said at least one intron enhances expression of said at least one heterologous DNA sequence.
A method of expressing a heterologous DNA sequence contained in an adenoviral vector, comprising operatively linking said heterologous DNA sequence with at least one intron selected from the group consisting of at least a portion of an intron of the Factor IX gene, the full seventh intron of the Factor IX gene, at least a portion of an intron of the Factor VIII gene and the first intron of the apolipoprotein A-1 gene, wherein said at least one intron enhances expression of said heterologous DNA sequence.
Dependencies are inferred from the claim text.