Abstract: Compositions and methods for detecting PRRG4- and PRRG2 associated molecules are provided as are methods of use thereof for the detection and treatment of cancer.

Abstract: The invention provides a method of preventing the formation of inhibitory antibodies in a mammal undergoing gene therapy. The method comprises administering to the mammal an immunosuppressive agent in conjunction with the gene therapy.

Abstract: Provided are compositions of viruses, the viruses including a recombinant adeno-associated virus vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding a blood coagulation factor and accompanying 5? and 3? untranslated regions and a transcription termination sequence.

Abstract: The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5? and 3? untranslated regions and a transcription termination.

Abstract: The invention provides compositions including modified blood clotting factors that have a non-native proteolytic cleavage site engineered into them allowing intracellular cleavage and secretion of an active form. The compositions are useful in the methods for treating a bleeding or clotting disorder.

Abstract: Compositions and methods are provided for inhibiting T cell mediated destruction of virally transduced, trangene containing cells.

Abstract: Compositions and methods for detecting PRRG4- and PRRG2 associated molecules are provided as are methods of use thereof for the detection and treatment of cancer.

Abstract: The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5? and 3? untranslated regions and a transcription termination

Abstract: Improved materials and methods for the treatment of Hemophilia A are provided.

Abstract: The invention relates to methods of optimizing gamma carboxylation of a vitamin K-dependent protein, methods of generating fully gamma carboxylated vitamin K-dependent protein, and compositions comprising chimeric nucleic acids and proteins for use in treatment of vitamin K-dependent disease states.

Abstract: Improved materials and methods for the treatment of Hemophilia A are provided.

Abstract: Methods for introducing recombinant adeno-associated virus (rAAV) virions into the liver of a mammal are provided. In these methods, the liver is partially or completely isolated from its blood supply, a catheter is introduced into the liver via a peripheral blood vessel, and rAAV virions are then infused through the catheter to the liver. The methods described herein may be used, for example, to deliver heterologous genes encoding therapeutic proteins to the hepatocytes of humans. This can be accomplished, for example, by introducing the catheter into a femoral artery, threading the catheter into the hepatic artery, and infusing rAAV virions through the catheter and into the liver. Exemplary examples of heterologous genes include those coding for blood coagulation factors.

Abstract: The present invention provides methods for administering recombinant adeno-associated virus (rAAV) virions to a human who has preexisting antibodies to wild-type adeno-associated virus (wtAAV) due to either a previous infection with wtAAV or to a previous administration of rAAV virions. In addition, the present invention also provides methods for treating hemophilia in a human who has preexisting antibodies to wtAAV or who has anti-rAAV antibodies, the methods involving administering rAAV virions that are rendered capable of expressing a heterologous gene that encodes for a blood coagulation factor whose expression results in a therapeutic benefit to the patient.

Abstract: The invention provides compositions including modified blood clotting factors that have a non-native proteolytic cleavage site engineered into them allowing intracellular cleavage and secretion of an active form. The compositions are useful in the methods for treating a bleeding or clotting disorder.

Abstract: Methods for introducing recombinant adeno-associated virus (rAAV) virions into the liver of a mammal are provided. In these methods, the liver is partially or completely isolated from its blood supply, a catheter is introduced into the liver via a peripheral blood vessel, and rAAV virions are then infused through the catheter to the liver. The methods described herein may be used, for example, to deliver heterologous genes encoding therapeutic proteins to the hepatocytes of humans. This can be accomplished, for example, by introducing the catheter into a femoral artery, threading the catheter into the hepatic artery, and infusing rAAV virions through the catheter and into the liver. Exemplary examples of heterologous genes include those coding for blood coagulation factors.

Abstract: Improved materials and methods for the treatment of Hemophilia A are provided.

Abstract: The invention relates to methods of optimizing gamma carboxylation of a vitamin K-dependent protein, methods of generating fully gamma carboxylated vitamin K-dependent protein, and compositions comprising chimeric nucleic acids and proteins for use in treatment of vitamin K-dependent disease states.

Abstract: Liver-directed gene transfer can induce immunological tolerance to a polypeptide associated with the expression of a therapeutic nucleic acid. Hepatic expression of a transgene induces tolerance to the expression product of the transgene, or to post-translational product related to transgene expression, thereby ameliorating or eliminating the immune responses associated with gene therapy and protein replacement, respectively, independent of the genetic background of the subject.

Abstract: The present invention provides methods for administering recombinant adeno-associated virus (rAAV) virions to a human who has preexisting antibodies to wild-type adeno-associated virus (wtAAV) due to either a previous infection with wtAAV or to a previous administration of rAAV virions. In addition, the present invention also provides methods for treating hemophilia in a human who has preexisting antibodies to wtAAV or who has anti-rAAV antibodies, the methods involving administering rAAV virions that are rendered capable of expressing a heterologous gene that encodes for a blood coagulation factor whose expression results in a therapeutic benefit to the patient.

Abstract: The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5' and 3' untranslated regions and a transcription termination signal, and methods of use thereof.