Abstract: The present invention relates to humanized monoclonal antibodies comprising the CDRs of murine antibody BMA031, which bind to the apTCR.CD3 complex and possess improved biological properties.

Abstract: The present invention improves significantly the success rate of lithotripsy and reduces the risk of tissue damage, by injecting a temporary plug in front, and optionally behind a concretion (for extracorporeal lithotripsy) or behind a concretion (for intracorporeal lithotripsy). One aspect of the present invention relates to injecting an inverse thermosensitive polymer solution into a lumen, thereby preventing the migration of a concretion, or its fragments, upon extracorporeal or intracorporeal lithotripsy.

Abstract: Polymers and compositions utilizing such polymers are disclosed for treating hyperphosphatemia and other illnesses associated with elevated serum phosphate levels. Phosphate binding polymers, or a pharmaceutically acceptable salt of the polymers, comprise pendent groups extending from a backbone of the polymer. Each pendent group comprises at least two nitrogen-bearing functional groups which bind phosphate. Variations of such polymer and compositions are disclosed.

Abstract: Provided are binding polypeptides (e.g., antibodies), and effector moiety conjugates thereof, comprising a CH1 domain (e.g., a human IgG1 CH1 domain), wherein the CH1 domain has an engineered N-linked glycosylation site at amino acid position 114, according to Kabat numbering. Also provided are nucleic acids encoding the antigen-binding polypeptides, recombinant expression vectors and host cells for making such antigen-binding polypeptides. Methods of using the antigen-binding polypeptides disclosed herein to treat disease are also provided.

Abstract: The present invention relates to methods of treating a subject with malaria comprising administering a 2-aminoindole compound represented by Formula: (I)—The values and preferred values of the variables in Structural Formula I are defined herein.

Abstract: The invention is directed to methods of producing a polypeptide or a variant thereof, wherein the polypeptide or variant thereof is dependent on LIMP-2 for trafficking, localization, stabilization and/or sorting of the polypeptide in the cell. In general, the methods comprise culturing a lysosomal integral membrane protein II (LIMP-2) deficient cell which expresses the polypeptide or the variant thereof under conditions in which the polypeptide or the variant thereof is produced.

Abstract: One aspect of the present invention relates to a method of ultrasonography, utilizing a gel comprising a reverse phase polymer which facilitates the transmission of high-frequency sound waves. Further, the inherent properties of the reverse phase polymer result in increased adhesion at higher temperatures, thereby helping to maintain the desired position of the ultrasound probe until the user intends to adjust the probe's position. In certain embodiments, the method is utilized in a medical procedure in which stability of an ultrasound probe or transducer in an intended desired position can improve the outcome or increase the efficiency of the procedure. In certain embodiments, the gel further comprises an additive to increase the ultimate adhesion of the gel. In still other embodiments, the gel can be used on the skin, on a protective sheath encasing an ultrasound probe, or between the sheath and the probe, or any or all of them.

Abstract: The disclosure provides methods for treating fatty liver disease and associated conditions by inhibiting the synthesis of glucosphingolipids, as exemplified by the use of glucosylceramide synthase substrate analogs.

Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.

Abstract: Novel methods for treating patients with autoimmune diseases are disclosed. The methods of the invention include first depleting circulating lymphocytes in the mammal, e.g., by administering anti-thymocyte antibody, and then, during the course of repopulation, administering to the mammal a therapeutically effective amount of latent TGF-? and/or another agent that promotes expansion of regulatory T cells. In certain aspects, the disclosed process results in improved kidney function and survival rates.

Abstract: The invention relates to isolation of novel ?-actin and ribosomal protein S21 (rpS21) promoters and uses thereof. In particular, this invention features nucleotide sequences for rodent ?-actin promoters including, hamster, rat, and mouse, and hamster rpS21 promoter.

Abstract: A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-?, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

Abstract: Provided herein are methods for the purification of recombinant adeno-associated virus (rAAV) vectors that can be used for gene transfer and specifically for gene therapy or vaccination. Recombinant AAV vectors of the invention are substantially free of in-process impurities, including production components such as cellular nucleic acids, cellular proteins, helper virus, and media components.

Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.

Abstract: The present invention provides compounds which are modulators of TNF-? signaling and methods of use thereof for treating a patient having a TNF-? mediated condition.

Abstract: Compounds, compositions and methods are provided for modulating the expression of apolipoprotein B. The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein B. Methods of using these compounds for modulation of apolipoprotein B expression and for diagnosis and treatment of diseases and conditions associated with expression of apolipoprotein B are provided.

Abstract: A method of treating polycystic kidney disease in a subject comprises administering to the subject an effective amount of a compound represented by Structural Formula (1): or a pharmaceutically acceptable salt thereof.

Abstract: The invention provides compositions and methods for treating breast cancer. Specifically, the invention relates to administering a Transforming Growth Factor beta (TGF?) antagonist in combination with capecitabine and ixabepilone to treat breast cancer.

Abstract: Methods of making ligand-decorated polymer conjugates of therapeutic glycoproteins are described. Improved targeting of glycoproteins to specific tissues is achieved by masking the natural carbohydrate and other surface determinants with high molecular weight polymers, such as, e.g., PEG, polysialic acid, etc., which in turn are decorated with target-specific ligands. In some embodiments, acid-labile linkages in such conjugates or rapidly degradable masking groups allow for the intracellular release of the polymer from the glycoprotein, for example, under conditions found in lysosomes.

Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.