Abstract: Methods for assaying function and/or activity and/or potency of isomerohydrolase proteins are provided.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

Abstract: The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

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 compositions and methods suitable for delivering RNAi agents against genetic targets in vascular and adjacent tissue in vivo so as to treat restenosis.

Abstract: AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.

Abstract: One form of a composition has two types of recombinant adeno-associated virus. The first type encodes a portion of Factor VIII operably linked to an expression control element; and the second type encodes a different portion of Factor VIII operably linked to an expression control element. The first and second nucleotide sequences collectively encode a functional Factor VIII protein. Another form of the composition is a recombinant adeno-associated virus containing a nucleotide sequence encoding functional Factor VIII light or heavy chain operably linked to a tissue-specific promoter.