Abstract: Compositions and methods for preventing, treating, or delaying the onset of rheumatoid arthritis involving 14-3-3?, are described. Provided is a method for preventing, treating, or delaying the onset of rheumatoid arthritis, the method comprising administering an effective amount of 14-3-3?, or a variant thereof, to a subject at risk for developing rheumatoid arthritis, and preventing, treating, or delaying the onset of rheumatoid arthritis in the subject.

Abstract: The present disclosure describes novel hybrid soluble ActRIIB-ECD polypeptides which fully retain binding affinity for myostatin and activin A but demonstrate significantly reduced binding to BMPs, especially BMP-9. The novel compositions described herein can be used to prepare novel hybrid ActRIIB ligand trap proteins, which can be used for modulating the growth of muscle, bone, cartilage, fat, fibroblast, blood and neuronal tissue to counteract muscle wasting, bone loss, anemia, inflammation and fibrosis in a therapeutically meaningful manner. Because these novel next-generation myostatin/activin inhibitors are safer and more effective molecules than the currently available myostatin inhibitors, they are useful in a wide variety of clinical indications.

Abstract: The invention provides a vaccine composition comprising a flavi peptide comprising one or more CD8+ T cell epitopes.

Abstract: The present invention relates to nicotinamide mononucleotide derivatives of Formula (I) for use in the treatment and/or prevention of a blood disorder, especially sickle cell disease. The present invention further relates to pharmaceutical compositions comprising compounds of Formula (I) for use in the treatment and/or prevention of a red blood cell disorder, especially sickle cell disease.

Abstract: The present disclosure relates to crystalline solids comprising a compound of Formula (I), wherein R is n-propyl, and methods of making compounds of Formula (I) wherein R is C1-C4 alkyl or C2-C4 alkenyl. The present disclosure also relates to crystalline solids comprising a compound of Formula (II), The present disclosure further relates to methods of preparing the crystalline solids, and pharmaceutical preparations of the crystalline solids, and use of such pharmaceutical preparations in treatment of diseases and conditions.

Abstract: The present invention relates to a polymerase enzyme from 9° N with improved ability to incorporate reversibly terminating nucleotides. The enzyme comprising mutations in the motif A region. The invention also relates to methods of using such enzymes as well as a kit with such polymerases.

Abstract: In one embodiment, the present invention provides a composition, wherein the composition is a porous scaffold, wherein the pores of the scaffold are from 1 to 500 microns, the composition comprising: a) a cross-linkable protein selected from the group consisting of collagen and gelatin; b) a cross-linker which induces cross-linking of the cross-linkable protein; and c) a liquid.

Abstract: A process for reducing the monosaccharide and/or disaccharide content in a food material, the process comprising contacting the food material with a glucosyltransferase that comprises an amino acid sequence having at least 95% identity to SEQ ID NO: 1.

Abstract: There are provided a pharmaceutical composition containing a lyophilized saccharide-degrading enzyme having excellent titer, and a package containing the pharmaceutical composition. The pharmaceutical composition is a unit dose formulation containing a lyophilized saccharide-degrading enzyme with a titer of not less than 0.3 unit/?g as an active ingredient, and containing the saccharide-degrading enzyme in an amount of not less than 2 ?g and not more than 8 ?g.

Abstract: Disclosed is a method for determining sensitivity to a CDK4/6 inhibitor, comprising the steps of: comparing a value based on activity of at least one CDK selected from CDK4 and CDK6 in a sample collected from a subject, with a threshold level corresponding to the CDK, and determining that the subject is insensitive to the CDK4/6 inhibitor when the value based on the CDK activity is less than the threshold level.

Abstract: Compositions and methods are provided for improved wound healing. In particular, provided herein are compositions and methods for the direct delivery of Sirtuin-1 (Sirt1) or vectors encoding Sirt1 to the wounds (e.g., of diabetic patients).

Abstract: A method, composition, and kit for treating a malarial infection in a subject by using an enzyme conjugate comprising a variant cystathione-gamma-lyase and a targeting ligand which binds to erythrocytes infected with Plasmodium pathogens. The variant cystathione-gamma-lyase has methioninase activity. Also disclosed is a method of treating Plasmodium-infected blood by exposing the infected blood with the enzyme conjugate.

Abstract: The invention relates to the use of a pharmaceutical composition containing nicotinamide (NAM) and/or pyruvate as a neuroprotective medicament or gene therapy in the treatment of neurodegenerative disorders, in particular axon degeneration of neuronal tissue in ocular-related neurodegeneration diseases including glaucoma.

Abstract: Systems and methods are provided for adjusting long acting insulin medicament dosages for a subject. A plurality of timestamped glucose measurements of the subject is obtained. A titration glucose level (246) is computed as a measure of central tendency (244, 268, 274) of a titration subset of small glucose measurements (240, 269, 273) identified within the timestamped glucose measurements, wherein the measure of central tendency represents a measure of small glucose measurements for the primary time window. A long acting insulin medicament dosage (216) is adjusted or maintained based upon the obtained titration glucose level (246).

Abstract: Disclosed herein are mesylate containing compositions, methods of use of such compositions and kits of components encompassing such compositions for improving the amplification of nucleic acids, especially in the presence of enzymatic inhibitors. These find particular use in forensic and environmental analyses. The instant disclosure provides new compositions, methods and kits of components for overcoming PCR inhibition. In some embodiments disclosed herein is a PCR master mix, the PCR master mix encompassing a mesylate.

Abstract: The present invention is related to arginine deiminase encapsulated into erythrocytes for use in therapy. It is in particular related to the use thereof in treating arginase-1 deficiency. Also, it relates to novel pharmaceutical compositions comprising arginine deiminase from M. arginini encapsulated into erythrocytes and the use thereof in treating diseases that may benefit from arginine depletion, such as arginine dependent cancers, in particular arginine-auxotrophic cancers, and arginase-1 deficiency.

Abstract: The present invention relates generally to isolated to arginine deiminase (ADI) proteins that have reduced cross-reactivity with anti-ADI-PEG 20 antibodies as compared to ADI-PEG 20 (pegylated ADI derived from M. hominis), but which can have functional characteristics comparable to or better than ADI-PEG 20, compositions comprising the ADI proteins, and related methods of treating arginine-dependent diseases or related diseases such as cancer.

Abstract: The invention includes, in part, methods and compounds for treating diseases and conditions characterized by elevated threonyl-tRNA synthetase (TARS) activity, which include, but are not limited to diseases and conditions in which angiogenesis is elevated as compared to normal. In some embodiments of the invention, a level of a TARS molecule is determined and compared to a control level of TARS to assess a treatment for a disease or condition characterized by elevated TARS activity.

Abstract: Pharmaceutical compositions comprising spray-dried oxalate decarboxylase crystals are disclosed. Methods to treat a disorder associated with elevated oxalate concentration using compositions comprising spray-dried oxalate decarboxylase crystals are also disclosed.

Abstract: The present invention relates to an EPRS (glutamyl-prolyl-tRNA synthetase) protein or a fragment thereof. The EPRS protein of the present invention or fragment thereof may bind to PCBP2 protein to activate the MAVS signaling pathway, and thus it has anti-RNA viral effects, thereby being effective for preventing or treating a RNA viral infectious disease.

Abstract: Provided is a therapeutic agent that effectively acts on a disease associated with abnormal glycosylation of dystroglycan. Also provided is a testing method for diseases associated with abnormal glycosylation of dystroglycan. Specifically, provided is a therapeutic agent for diseases associated with abnormal glycosylation of dystroglycan, containing CDP-ribitol as an active ingredient. Ribitol-phosphate is important in the glycan structure of dystroglycan. In order for ribitol-phosphate to be incorporated into a dystroglycan glycan, a material therefor (sugar donor) is required. In the present invention, it has been found for the first time that CDP-ribitol serves as the sugar donor. It has been confirmed that the glycan of ISPD-deficient cells can be restored by administering CDP-ribitol. Thus, the present invention, which allows CDP-ribitol to be utilized for supplementation therapy, has been completed.

Abstract: Methods and compositions related to the engineering of a protein with L-cyst(e)ine degrading enzyme activity are described. For example, disclosed are modified cystathionine-?-lyases comprising one or more amino acid substitutions and capable of degrading L-cyst(e)ine. Furthermore, compositions and methods are provided for the treatment of cancer and cystinuria using the disclosed modified enzymes or nucleic acids encoding said enzymes.

Abstract: Disclosed herein are compositions and methods for treating Type II diabetes. The compositions comprise plant expressed Exendin 4. Particularly exemplified are plant derived compositions that include a CTB-EX4 conjugate that is bioencapsulated in chloroplasts.

Abstract: The present invention relates to molecules which function as modulators (i.e., inhibitors and agonists) of the Ras-homologous (Rho) family of small GTPases (e.g. Rac, Cdc42 and Rho GTPases) and their use to treat diseases, including cancers (including solid tumors-medulloblastoma, ovarian, breast, head and neck, testicular, prostate among others and hematologic malignancies-B cell lymphoma, where these GTPases are overexpressed or hyperactivated), sporadic and genetic diseases where activation of Rho GTPases plays a pivotal role (Menkes disease, rheumatoid arthritis, atherosclerosis, diabetes (type I), Huntington's disease and Alzheimer's disease) which are mediated through these proteins. Compounds according to the present invention may also be used as a therapy for the treatment of Entamoeba spp. or Acanthamoeba spp. infections, especially including Entamoeba histolytica.

Abstract: The present invention relates to a composition for preventing or treating sepsis or septic shock including a Mycobacterium tuberculosis-derived adenosine kinase (ADK) protein as an active ingredient. The Mycobacterium tuberculosis-derived ADK protein according to one exemplary embodiment of the present invention has an effect of inhibit binding between the LPS and cells by binding to LPS, and also has an excellent therapeutic effect against sepsis by enhancing viability in a sepsis animal model and suppressing inflammatory response in the lung and cell death in the spleen.

Abstract: Compositions and methods are provided for improved wound healing. In particular, provided herein are compositions and methods for the direct delivery of Sirtuin-1 (Sirt1) or vectors encoding Sirt1 to the wounds (e.g., of diabetic patients). In some embodiments, provided herein are therapeutic devices comprising: (a) a vector encoding Sirtuin-1 (Sirt 1); and (b) a hydrogel carrier. In some embodiments, the vector comprises a viral vector comprising a polynucleotide sequence encoding Sirt 1. In some embodiments, the vector comprises a non-viral vector comprising a polynucleotide sequence encoding Sirt1.

Abstract: Disclosed herein are compositions and methods for treating Type II diabetes. The compositions comprise plant expressed Exendin 4. Particularly exemplified are plant derived compositions that include a CTB-EX4 conjugate that is bioencapsulated in chloroplasts.

Abstract: A polypeptide having chemokine activity mediated by binding to C-C chemokine receptor type 3 (CCR3) comprising SEQ ID NO: 1 and uses thereof are described.

Abstract: Methods for design of genetic circuits are provided.

Abstract: Disclosed herein are compositions and formulations containing a TATk-CDKL5 fusion protein. Also disclosed are methods of producing a TATk-CDKL5 fusion protein from vectors containing a TATk-CDKL5 cDNA and methods of transducing cells with the vectors containing a TATk-CDKL5 cDNA and the TATk-CDKL5 fusion protein.

Abstract: Pharmaceutical compositions comprising spray-dried oxalate decarboxylase crystals are disclosed. Methods to treat a disorder associated with elevated oxalate concentration using compositions comprising spray-dried oxalate decarboxylase crystals are also disclosed.

Abstract: A method of treating demyelinating diseases involves administering a PAK1 inhibitor that is PF-3758309 to a subject in need of such treatment.

Abstract: Provided herein are compositions containing a lysosomal storage disorder replacement enzyme (LSDRE) and a dispersing agent for subcutaneous injection for treatment of lysosomal storage diseases. Kits and methods of treatment are also provided.

Abstract: Human cystathionine ?-synthase variants are disclosed, as well as a method to produce recombinant human cystathionine ?-synthase and variants thereof. More particularly, the role of both the N-terminal and C-terminal regions of human CBS has been studied, and a variety of truncation mutants and modified CBS homologs are described. In addition, a method to express and purify recombinant human cystathionine ?-synthase (CBS) and variants thereof which have only one or two additional amino acid residues at the N-terminus are described.

Abstract: Pharmaceutical compositions comprising spray-dried oxalate decarboxylase crystals are disclosed. Methods to treat a disorder associated with elevated oxalate concentration using compositions comprising spray-dried oxalate decarboxylase crystals are also disclosed.

Abstract: A method of treating a disease selected from the group consisting of emphysema, sepsis, septic shock, ischemic injury, cerebral ischemia, a neurodegenerative disorder, meningitis, encephalitis, hemorrhage, cerebral ischemia, heart ischemia and a cognitive deficit in a subject in need thereof is provided. The method comprising administering to the subject a therapeutically effective amount of a combination of at least two agents, wherein a first of said two agents upregulates an activity and/or expression of Nrf2 and a second of said two agents is a glutamatergic modulator, thereby treating the disease.

Abstract: The present invention provides PiggyBac transposase proteins, nucleic acids encoding the same, compositions comprising the same, kits comprising the same, non-human transgenic animals comprising the same, and methods of using the same.

Abstract: The invention includes, in part, methods and compounds for treating diseases and conditions characterized by elevated threonyl-tRNA synthetase (TARS) activity, which include, but are not limited to diseases and conditions in which angiogenesis is elevated as compared to normal. In some embodiments of the invention, a level of a TARS molecule is determined and compared to a control level of TARS to assess a treatment for a disease or condition characterized by elevated TARS activity.

Abstract: The present disclosure relates to protein and nucleic acid mutants of chondroitinase ABCI. Such nucleic acid mutants encode for chondroitinase ABCI mutant enzymes exhibiting altered chondroitin lyase activity or increased resistance to inactivation from stressors including UV light or heat. Methods of using such nucleic acid mutants encoding chondroitinase ABCI mutant enzymes is also provided.

Abstract: Disclosed herein are a modified sulfamidase, a composition comprising a modified sulfamidase, as well as methods for preparing a modified sulfamidase and therapeutic use of such a sulfamidase. In particular, the present disclosure relates to a modified sulfamidase comprising substantially no epitopes for glycan recognition receptors, thereby enabling transportation of said sulfamidase across the blood brain barrier of a mammal, wherein said sulfamidase has catalytic activity in the brain of said mammal.

Abstract: Disclosed herein are chimeric polypeptides, including compositions thereof, expression vectors, and methods of use thereof, for the generation of transgenic cells, tissues, plants, and animals. The compositions, vectors, and methods of the present invention are also useful in gene therapy techniques. The invention provides a chimeric polypeptide. The polypeptide includes: a) a recombinase, nuclease or transcription factor, or fragment thereof; and b) a transcription activator-like effector (TALE) protein.

Abstract: The application concerns means for predicting whether a subject infected with one or more HCVs has a high probability of responding to an anti-HCV treatment which will comprise the administration of interferon and of ribavirin or whether, in contrast, that subject has a high probability of not responding to that anti-HCV treatment. The means of the invention in particular involve assaying the levels of expression of selected genes, said selected genes being: at least one gene from among MBL2, LGALS3BP and IL8, and at least one gene from among G1P2, CCL21 and CXCL10, and optionally, at least one gene from among AFP, CRP, CXCL11, CXCL6, CXCL9, FGF7, MDK, MMP2, SFN, TGFB2 and VEGFD.

Abstract: Provided are methods for screening a subject for cancer. The methods involve obtaining a blood sample from the subject and determining a level of Bridging Integrator 1 (BIN1) isoforms comprising exon 12a in the sample. Optionally, the method involves determining a level of 12a+/13? BIN isoform (comprising exon 12a but lacking exon 13) in the sample. An elevated level of 12a+ (e.g., 12a+/13?) BIN1 isoforms in the blood sample indicates the subject has cancer. Also provided are methods for determining efficacy of a cancer therapy in a subject and methods of treating cancer. Isolated antibodies that selectively bind human 12a+ BIN1 are also provided as well as kits for determining 12a+/13? BIN1 isoforms.

Abstract: The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transposon, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.

Abstract: One aspect of the present invention relates to mutants of chondroitinase ABCI. Such chondroitinase ABCI mutants exhibit altered chondroitin lyase activity or increased resistance to inactivation from stressors including exposure to UV light or heat. Methods of using chondroitinase ABCI mutant enzymes are also provided.

Abstract: Provided herein are methods for ameliorating the pathophysiology cysteine protease exotoxin comprising the step of administering to said individual an effective dose of an S-nitrosylating agent and an inositol phosphate or analog thereof.

Abstract: The invention provides a process for the purification recombinantly expressed, self-assembled VLP from the homogenate of a bacterial host, wherein the process can be scaled up to a commercial production scale in a cost effective manner. The process comprises a first chromatography using an anion exchange matrix, a second chromatography using hydroxyapatite and, optionally, a size exclusion chromatography. VLP preparations obtained by the process of the invention are essentially free of endotoxin contaminations.

Abstract: Human cystathionine ?-synthase variants are disclosed, as well as a method to produce recombinant human cystathionine ?-synthase and variants thereof. More particularly, the role of both the N-terminal and C-terminal regions of human CBS has been studied, and a variety of truncation mutants and modified CBS homologues are described. In addition, a method to express and purify recombinant human cystathionine ?-synthase (CBS) and variants thereof which have only one or two additional amino acid residues at the N-terminus are described.

Abstract: The invention provides methods for overriding cell cycle arrest in a tumor cell, which comprise inducing DNA damage in the cell, and contacting the cell with an amount of bosutinib or a bosutinib isomer effective to inhibit one or more kinase constituents of a DNA damage checkpoint pathway. The invention also provides novel bosutinib isomers, as well as compositions of the novel isomers and the bosutinib isomer 3,5-dichloro-4-methoxyaniline.

Abstract: The present disclosure provides cell culture medium comprising trimethylamine N-oxide (TMAO). Progenitor cells cultured with such medium can form connective tissue with enhanced mechanical properties. Also provided are methods of forming connective tissue and methods of treatment for connective tissue defects.