Abstract: The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 and related AAV vectors target polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences.

Abstract: The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 and related AAV vectors target polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences.

Abstract: Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use.

Abstract: The disclosure of the present application provides compositions and methods for treating a blood disorder. In at least one embodiment of a method for treating a patient with a plasma protein deficiency disorder, the method comprises the steps of administering a cell-based composition to a patient with a plasma protein deficiency disorder to treat the plasma protein deficiency disorder, where the cell-based composition comprises a mammalian adipose stromal cell that is capable of effectuating the production of a plasma protein within the patient.

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.