Abstract: This application provides methods of improving protein replacement therapy by combining protein replacement therapy with active site-specific chaperones (ASSC) to increase the stability and efficiency of the protein being administered. The application further provides stable compositions comprising the purified protein and an ASSC, and methods of treatment by administering the compositions.

Abstract: The present invention relates to methods of constructing an integrated artificial immune system that comprises appropriate in vitro cellular and tissue constructs or their equivalents to mimic the normal tissues that interact with vaccines in mammals. The artificial immune system can be used to test the efficacy of vaccine candidates in vitro and thus, is useful to accelerate vaccine development and testing drug and chemical interaction with the immune system.

Abstract: This application provides methods of improving protein replacement therapy by combining protein replacement therapy with active site-specific chaperones (ASSC) to increase the stability and efficiency of the protein being administered. The application further provides stable compositions comprising the purified protein and an ASSC, and methods of treatment by administering the compositions.

Abstract: The invention provides a method for generating a transgenic eukaryotic cell population having a modified human Rosa26 locus, which method includes introducing a functional DNA sequence into the human Rosa26 locus of starting eukaryotic cells. Also provided are targeting vectors useful in the method, as well as a cell population and a transgenic non-human animal comprising a modified human Rosa26 locus. Finally, the invention provides an isolated DNA sequence corresponding to the human Rosa26 locus.

Abstract: Method for enhancing in a mammalian cell the activity of an enzyme associated with Gaucher Disease by administering a competitive inhibitor of glucocerebrosidase in an amount effective to enhance the activity of the enzyme. Preferred compounds for use in the method are imino sugars and related compounds. In particular, C8-12-alkyl derivatives of N-alkyl-deoxynojirimycin, isofagomine compounds, and calystegine compounds are effective to enhance glucocerebrosidase activity.

Abstract: The present invention relates to methods of constructing an integrated artificial immune system that comprises appropriate in vitro cellular and tissue constructs or their equivalents to mimic the normal tissues that interact with vaccines in mammals. The artificial immune system can be used to test the efficacy of vaccine candidates in vitro and thus, is useful to accelerate vaccine development and testing drug and chemical interaction with the immune system.

Abstract: A method of enhancing the activity of lysosomal ?-Galactosidase A (?-Gal A) in mammalian cells and for treatment of Fabry disease by administration of 1-deoxy-galactonojirimycin and related compounds.

Abstract: A method of enhancing the activity of lysosomal ?-Galactosidase A (?-Gal A) in mammalian cells and for treatment of Fabry disease by administration of 1-deoxy-galactonojirimycin and related compounds.

Abstract: Disclosed herein is a novel method of treating vitiligo by using an excimer laser that emits light in the UVB range. The invention includes a method of incrementally increasing exposure of affected vitiligo areas with UVB laser light from an excimer laser to restore pigmentation to skin areas afflicted with vitiligo.

Abstract: Method for enhancing in a mammalian cell the activity of an enzyme associated with Gaucher Disease by administering a competitive inhibitor of glucocerebrosidase in an amount effective to enhance the activity of the enzyme. Preferred compounds for use in the method are imino sugars and related compounds. In particular, C8-12-alkyl derivatives of N-alkyl-deoxynojirimycin, isofagomine compounds, and calystegine compounds are effective to enhance glucocerebrosidase activity.

Abstract: The invention discloses compositions and methods for decreasing osteoclast which are useful for the treatment of a variety of bone loss disorders.

Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.

Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.

Abstract: Disclosed are methods of identifying and diagnosing certain types of cancers and pre-stages thereof in a patient by identifying alternatively spliced isoforms of wild type KLF6 (KLF6wt), in particular any one of the isoforms selected from the group consisting of: KLF6 splice variant-1 (KLF6SV1), KLF6 splice variant-2 (KLF6SV2), and KLF6 splice variant-3 (KLF6SV3). Also disclosed are methods for diagnosing cancer using the polypeptides and polynucleotides identified herein, as well as methods of treating certain types of cancers by inhibiting polynucleotides and polypeptides identified herein.

Abstract: The invention relates to a glucan having a beta-(1,3)-backbone with one or more beta-(1,3)-side chains linked thereto for use in the treatment of asthma and related diseases of abnormal pulmonary function in an animal. Also described is a method of treating asthma and related diseases of abnormal pulmonary function in an animal comprising administering to said animal an effective amount of a glucan having a beta-(1,3)-backbone with one or more beta-(1,3)-side chains linked thereto.

Abstract: Methods, systems and apparatus for characterizing networks are presented. For example, a method of characterizing a network represented by a plurality of nodes and a plurality of edges is provided. The method may be implemented on a processor device and includes calculating, for example, by the processor device, a passthrough count of at least a portion of the network. The passthrough count includes a count of a number of passthroughs in the at least a portion of the network. A passthrough includes one of the plurality of nodes, a directed edge of the plurality of edges coupled to the one of the plurality of nodes, and another edge of the plurality of edges coupled to the one of the plurality of nodes. At most one of the directed edge and the other edge is directed towards the one of the plurality of nodes. At most one of the directed edge and the other edge is directed away from the one of the plurality of nodes.

Abstract: Methods to improve the tropism or other features of a virus are disclosed. Such methods can be used to prepare, e.g., DNA or plasmid libraries of variants of a gene encoding a viral capsid or envelope protein having a randomly inserted restriction site, libraries of viral clones with such variant genes with a randomly inserted restriction site or polypeptide sequence targeting a receptor expressed by a specific type of mammalian cells. Described are also methods to prepare mosaic viruses, i.e., viral particles wherein copies of one or more capsid or envelope proteins originate from different sources. These methods can be used to prepare mosaic viruses of a specific mixture of wild-type and mutant proteins, or of different types of mutant proteins.

Abstract: The present invention provides methods and compositions for treating or preventing allergic reactions, particularly anaphylactic reactions. Methods of the present invention involve administering microorganisms to allergic subjects, where the microorganisms contain a recombinant version of the protein allergen. The recombinant version can be wild-type or may include mutations within IgE epitopes of the protein allergen. Preferably the compositions are administered rectally. Particularly preferred microorganisms are bacteria such as E. coli. Any allergen may be used in the inventive methods. Particularly preferred allergens are anaphylactic allergens including protein allergens found in foods, venoms, drugs and latex. The inventive compositions and methods are demonstrated in the treatment of peanut-induced anaphylaxis.

Abstract: The disclosure provides methods for treating or preventing a cancerous condition, such as multiple myeloma, by administering a therapeutically effective combination of a proteasome inhibitor and salubrinal.

Abstract: The present invention provides cell populations that are enriched for mesendoderm and mesoderm, and cell populations that are enriched for endoderm. The cell populations of the invention are useful for generating cells for cell replacement therapy.