The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.

The invention relates to a method for purifying biologically active GLA-domain coagulation proteins, comprising the following steps: a) bringing a sample that contains one or more GLA-domain coagulation proteins and may contain biologically inactive molecules of GLA-domain protein(s), into contact with an affinity support on which nucleic aptamers that bind specifically to at least one biologically active GLA-domain coagulation protein are immobilized, in order to form complexes between (i) said nucleic aptamers and (ii) said GLA-domain coagulation protein(s), b) releasing the GLA-domain coagulation protein(s) from the complexes formed in step a), and c) recovering said biologically active GLA-domain coagulation protein(s) in a purified form.

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.

Methods of isolating highly sialylated recombinant vitamin K dependent proteins, particularly Factor IX, by chromatographic methods are described. The highly sialylated recombinant proteins are characterized. The improved Factor IX has at least 62% N-glycosylation with 3 or 4 sialic acid residues and improved bioavailability and pharmokinetic properties.

Disclosed herein are compositions and methods for modulatimi 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.

Disclosed herein are recombinant viral vectors comprising a liver specific promotor in operable combination with a heterologous nucleic acid sequence encoding a protein, such as a clotting factor. Methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B, are also provided.

Methods, systems and computer program products for shared content management systems that provide performance analytics pertaining to a project. Embodiments include establishing one or more network communication links between a content management system that manages a plurality of shared content objects and a plurality of applications that cause modifications to the shared content objects in accordance with workflows of the project. Iteraction events that correspond to modifications over the shared content objects are recorded such that interaction events associated with the plurality of applications are selected based at least in part on attributes associated with the interaction events. Relationships between the recorded interaction events such as time durations between certain of the interaction events are calculated. Project performance measurements are generated based on the calculations and/or based on other relationships between the interaction events. The calculations may span across many different applications and/or many different departments and/or many different enterprises.

Provided is a platform for expressing a protein of interest by artificially manipulating the liver, and more particularly, to a platform for alleviating or treating a genetic disorder or improving a body function by inducing expression by inserting a transgene (e.g., a therapeutic gene) which can function or be expressed normally, into a high-expression secretory gene, instead of a disease gene which functions or is expressed abnormally. The high-expression secretory gene includes the HP or APOC3 gene. The transgene includes one that is highly expressed using a promoter in a hepatocyte genome and is secretory out of the cell.

The present invention relates to compositions comprising factor IX coagulation factors linked to extended recombinant polypeptide (XTEN), isolated nucleic acids encoding the compositions and vectors and host cells containing the same, and methods of making and using such compositions in treatment of coagulation factor-related diseases, disorders, and conditions.

The present invention provides a therapeutic agent for epithelial and endothelial injury, and in particular, for epithelial and endothelial microinjury, and the like. The therapeutic agent according to the present invention comprises, for example, a peptide of the following (a), (b), etc., a derivative thereof, or their salt: (a) a peptide comprising the amino acid sequence shown in any one of SEQ ID NOS: 10, 4, 12 and 6; or (b) a peptide comprising the amino acid sequence shown in any one of SEQ ID NOS: 16, 18, 20 and 22.