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AssigneeChiron Corporation
InventorsArun Srivastava, Selvarangan Ponnazhagan, Robert H. Schloemer, Xu-Shan Wang, Mervin C. Yoder, Shang-Zhen Zhou, Jaime Escobedo, Varavani Dwarki
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Provided are methods for selectively expressing therapeutic molecules, such as secretory proteins, antisense molecules and ribozymes, in the liver. The methods find use in treating hepatic diseases or conditions. The methods also find use in treating any disease or condition in which systemic administration of the therapeutic substance, for example, a secretory protein, is desired. The methods involve administering to a mammalian patient having a need for liver expression of a therapeutic molecule an AAV vector containing a therapeutically effective amount of the therapeutic molecule. Also provided are novel vectors employable in these methods.
A method for realizing liver specific delivery of a therapeutic molecule in a mammalian patient comprising administering to said mammalian patient a therapeutically effective amount of an AAV vector containing said therapeutic molecule.
A hybrid helper AAV vector comprising a DNA sequence encoding hepatitis B virus surface antigen or a functional fragment thereof linked to a DNA sequence encoding the AAV VP-1 protein to form a chimeric DNA sequence.
A recombinant AAV vector comprising two AAV ITRs (inverted terminal repeats) in which the native D-sequences of each of said ITRs are modified by the substitution of nucleotides such that at least 5 native nucleotides and up to 18 native nucleotides are retained and the remaining nucleotides of the D-sequence are deleted or replaced with non-native nucleotides.
A recombinant AAV vector comprising two AAV ITRs (inverted terminal repeats) in which the native D-sequences of each of said ITRs are modified by the substitution of nucleotides such that at least 10 native nucleotides up to 18 native nucleotides are retained and the remaining nucleotides of the D-sequence are deleted or replaced with non-native nucleotides.
A recombinant AAV vector comprising two AAV ITRs (inverted terminal repeats) in which the native D-sequences of each of said ITRs are modified by the substitution of nucleotides such that 10 native nucleotides are retained and the remaining nucleotides of the D-sequence are deleted or replaced with non-native nucleotides.
A recombinant AAV vector selected from the group consisting of pD-5, pD-10, pD-15 and pD-20.
Dependencies are inferred from the claim text.