Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.

An antibody, preferably a blocking antibody, and most preferably a monoclonal antibody (mAb), specific for human endothelial cell protein C receptor (EPCR) such as mAbs JRK 1494 or JRK 1535 is used to reduce or attenuate joint swelling, macrophage infiltration, iron deposition and/or blood vessel formation and to treat arthropathy in a hemophilic subject.

An antibody, preferably a blocking antibody, and most preferably a monoclonal antibody (mAb), specific for human endothelial cell protein C receptor (EPCR) such as mAbs JRK 1494 or JRK 1535 is used to reduce or attenuate joint swelling, macrophage infiltration, iron deposition and/or blood vessel formation and to treat arthropathy in a hemophilic subject.

Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.

The invention relates to a chimeric protein comprising at least one clotting factor and at least a portion of an immunoglobulin constant region. The invention relates to a method of treating a hemostatic disorder comprising administering a therapeutically effective amount of a chimeric protein wherein the chimeric protein comprises at least one clotting factor and at least a portion of an immunoglobulin constant region.

Disclosed are a modified FIX (Factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.

Polypeptides comprising at least one carboxy-terminal peptide (CTP) of chorionic gonadotropin attached to the carboxy terminus but not to the amino terminus of a coagulation factor and polynucleotides encoding the same are disclosed. Pharmaceutical compositions and pharmaceutical formulations comprising the polypeptides and polynucleotides of the disclosure and methods of using and producing same are also disclosed.

Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.