Abstract: The disclosure provides a correlation between the expression level of the miR-193a gene, which can be regulated by its methylation status, and both tumorigenesis of and the resistance of a cancer cell to a pyrimidine antimetabolite (5-FU) based chemotherapy. In addition to the methylation status and the expression of miR-193a, its downstream genes, such as E2F1, SRSF2, and apoptotic genes such as caspase 2, are also involved and can serve as useful markers for cancer therapy prognosis and for therapy selection.

Abstract: The present invention relates to a process for purification of recombinant alpha-mannosidase, a process for production of alpha-mannosidase, a composition comprising alpha-mannosidase, use of the composition as a medicament, use as a medicament for the treatment of alpha-mannosidosis and a method of treating alpha-mannosidosis and/or alleviating the symptoms of alpha-mannosidosis.

Abstract: Prodrugs or pharmaceutically acceptable salts, stereoisomers, solvates, or polymorphs thereof include a pharmaceutically and/or diagnostically active cationic steroidal antimicrobial (hereinafter “CSA”) compound or pharmaceutically acceptable salt, stereoisomer, solvate, or polymorph thereof, and one or more cleavable groups (C.G.). Some embodiments include a CSA compound prepared in an inactive or less active form and that is capable of conversion to a fully active form upon administration to a subject, upon preparation of a pharmaceutical formulation containing the CSA composition, and/or upon exposure to physiological conditions. Pharmaceutical compositions include the prodrug or pharmaceutically acceptable salt, stereoisomer, solvate, or polymorph thereof and one or more pharmaceutically acceptable excipients.

Abstract: Aspects of the present invention relate to peptides having anti-inflammatory activity, compositions containing one or more of the peptides, and use of the peptides to treat conditions associated with excessive inflammation in animals, particularly humans and other mammals.

Abstract: Treatment of tumors, especially breast cancer or glioblastoma tumors, by silencing RAB27A and/or TRAF3IP2, compositions and methods for same.

Abstract: This invention relates to the use of CRISPR nucleic acids to screen for essential and non-essential genes and expendable genomic islands in bacteria, archaea, algae and/or yeast, to kill bacteria, archaea, algae and/or yeast, to identify the phenotype of a gene or genes, and/or to screen for reduced genome size and/or a gene deletion in bacteria, archaea, algae and/or yeast.

Abstract: Methods and systems capturing particles suspended in a fluid flowed through a micro-channel, can include flowing the fluid including the particles to be captured through a micro-channel and past a groove defined in a surface of a wall of the micro-channel such that flowing the fluid past the groove forms microvortices in the fluid; contacting at least some of the particles against an adherent disposed on one or more of walls of the microchannel after the microvortices form in the fluid; and capturing at least some of the particles contacting the adherent.

Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of UTRN. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of UTRN. Methods for modulating expression of UTRN using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of UTRN.

Abstract: The present invention relates to a factor for reprogramming bone endothelial cells to promote bone angiogenesis and osteogenesis as well as to an ex vivo method for reprogramming a bone endothelial cell to achieve cells able to mediate bone angiogenesis and osteogenesis. The subtype of bone endothelial cells mediating bone angiogenesis and osteogenesis express CD31 and Endomucin as markers.

Abstract: In one aspect, the present invention relates to a mammalian cell-based high-throughput assay for the profiling and screening of human epithelial sodium channel (hENaC) cloned from a human kidney c-DNA library and is also expressed in other tissues including human taste tissue. The present invention further relates to amphibian oocyte-based medium-throughput electrophysiological assays for identifying human ENaC modulators, preferably ENaC enhancers. Compounds that modulate ENaC function in a cell-based ENaC assay are expected to affect salty taste in humans.

Abstract: There is provided methods of determining tuberculosis (TB) infection status in an individual comprising: (i) providing a sample comprising T-cells; (ii) exposing the sample of (i) to one or more TB antigens; (iii) identifying T-cells in the sample that are CD4 positive and (a) secrete TNF-? without secreting IFN-?; or (b) secrete IFN-? without secreting TNF-?; (iv) identifying those cells of (iii) which are also CCR7 and, CD127 negative; and optionally (v) calculating the cells identified in (iv) as a percentage of those identified in (iii); wherein the identification of cells in (iv) and/or the percentage of T-cells calculated in (v) correlates to TB infection status of the individual, and wherein steps (iii) and (iv) can be carried out either sequentially or simultaneously. There are also provided compositions and kits for use in such methods.

Abstract: The present invention is directed towards methods of culturing non-keratinocyte epithelial cells, with the methods comprising culturing non-keratinocyte epithelial cells in the presence of feeder cells and a calcium-containing medium while inhibiting the activity of Rho kinase (ROCK) in the feeder cell, the non-keratinocyte epithelial cells or both during culturing.

Abstract: The present invention relates to a medium composition for enhancing the self-renewal ability of stem cells, which contains apelin, and a method of culturing stem cells using the medium composition. According to the present invention, stem cells can be effectively cultured to proliferate without changing the characteristics of the cells, and the telomerase activity of stem cells can be increased. Thus, the efficacy of cell therapy based on stem cells can be significantly improved.

Abstract: Bruton's tyrosine kinase (Btk) plays an important role in the pathophysiology of local inflammation in acute lung injury (ALI)/acute respiratory distress syndrome (ARDS). A unique two hit model of ALI induced by lipopolysaccharide (LPS)/immune complex (IC)—was developed, along with viral-induced ALI caused by influenza virus and COPD caused by second hand smoke. Two types or therapeutics (i) Anti-neutrophil antibodies, and specifically their F(ab?)2 fragments conjugated to Btk-specific siRNA or (ii) small molecule Btk inhibitors were administered to lungs and silenced or inhibited Btk specifically in alveolar neutrophils. Such silencing/inhibition had a dramatic protective effect lung damage induced by LPS/IC, influenza virus and on COPD. Btk regulates neutrophil survival and clearance of apoptotic neutrophils in these diseases. Btk-targeted neutrophil-specific therapy restores homeostasis in lungs of patients with ALI/ARDS, acute lung infection, COPD and other lung diseases.

Abstract: The present invention relates to compositions and methods of inhibiting stem cell binding to organs and tissues, including the blocking of stem cell binding to germinal centers present in lymph tissue. Disclosed are compositions and methods for regenerating germinal centers in lymphatic tissue. Included in the compositions are adjuvants, agonists to CD40, CD28 and the IL-21 receptor, and antagonist to CD20.

Abstract: The present invention is directed towards methods of culturing non-keratinocyte epithelial cells, with the methods comprising culturing non-keratinocyte epithelial cells in the presence of feeder cells and a calcium-containing medium while inhibiting the activity of Rho kinase (ROCK) in the feeder cell, the non-keratinocyte epithelial cells or both during culturing.

Abstract: The present application relates to nucleic acid compounds, compositions comprising same and methods of use thereof for treatment of various diseases, disorders and conditions. The compounds are preferably chemically synthesized and modified double-stranded nucleic acid molecules which down regulate expression of a p53 gene.

Abstract: RNA interference (RNAi) agents and RNAi agent conjugates for inhibiting the expression of the LPA (apo(a)) gene are described. Pharmaceutical compositions comprising one or more LPA RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described LPA RNAi agents to liver cells in vivo provides for inhibition of LPA gene expression and treatment of cardiovascular and cardiovascular-related diseases.

Abstract: The present invention relates to method, system, and composition for modulating the compliance of the trabecular meshwork, which may provide treatment to glaucoma.

Abstract: The purpose of the present invention is to provide a composition for effectively inducing apoptosis and/or proliferation inhibition in a cell, and a method using the same. An agent for inducing apoptosis that comprises as active ingredients a drug inhibiting GST-? and a drug inhibiting Akt; a medicinal composition comprising the same; a method for treating a disease caused by abnormality in apoptosis using the same, etc.

Abstract: The polymeric carrier for delivering nucleic acid material to a cell is provided herein. The polymeric carrier can include a dendrimer group having 2 to 8 termini, each of the termini having an arginine-grafted bioreducible polymer attached thereto. In one embodiment, only a portion of the termini can have an arginine-grafted bioreducible polymer attached thereto.

Abstract: Provided herein are methods of treating and preventing pancreatitis, such as pancreatitis induced by glucagon-like peptide (GLP) agonists (such as GLP-1 agonists, for example Byetta®), by administration of a vitamin D receptor agonist (such as vitamin D, vitamin D analogs, vitamin D precursors, and vitamin D receptor agonists precursors). In some examples the subject has diabetes, such as type 2 diabetes.

Abstract: The present invention provides a method for inducing differentiation of a human pluripotent stem cell into an intermediate mesoderm cell without the use of a growth factor. Such method comprises performing culture in a medium containing a specific compound, so as to induce differentiation of a human pluripotent stem cell into an intermediate mesoderm cell.

Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

Abstract: The purpose of the present invention is to provide an evaluation method kit for evaluating the risk of onset of diffuse alveolar damage due to such factors as anticancer drug administration. The purpose of the present invention is also to provide a method for evaluating the risk of onset of side effects in anticancer drug treatment. An evaluation method and evaluation kit for evaluating the risk of onset of diffuse alveolar damage, including detecting gene polymorphism present in the MUC4 gene. A method for evaluating the risk of onset of side effect in anticancer drug treatment, including detecting gene polymorphism present in the MUC4 gene of a patient scheduled for anticancer drug administration.

Abstract: The invention relates to novel fluorescent dyes based on the following pyrenyloxy sulfonamide structure: wherein R1 is a leash joined to the pyrenyloxy group via an ether link containing generally a reactive functional group such as, activated carbonate, activated ester, amino group, azide or alkyne for conjugation with biomolecules; R2 and R3 are hydrogen atoms, or short alkyl chains, or cyclic rings with or without heteroatoms such as nitrogen, oxygen, sulfur, phosphorus. The spectral properties of the fluorescent dyes are sufficiently different in wave-lengths and intensity from fluorescein as to permit simultaneous use of fluorescein and/or more other fluorescent dyes with minimum interference and to avoid interference from endogenous green fluorescent protein in biological system. The dyes are non-ionic to facilitate their entry into cells for intracellular detection. The non-ionic structure also precludes undesired electrostatic reactions with ionic sites on biological components and structures.

Abstract: The present invention relates to the mannose-receptor selective lysinylated cationic amphiphile and a process for preparation thereof. The compounds of the present invention can target DNA vaccines to antigen presenting cells (APCs) such as macrophages and dendritic cells (DCs), via mannose receptors expressed on the cell surface of APCs. The cationic amphiphiles disclosed herein show enhanced cellular and humoral immune response compared to their mannosyl counterparts in genetic immunization in mice. The present invention discloses that immunization with electrostatic complexes (lipoplexes) of DNA vaccines encoding melanoma antigens (gp100 and tyrosinase) and liposome of the presently described novel lysinylated cationic amphiphiles with mannose-mimicking shikimoyl head-groups provides long-lasting (100 days post melanoma tumor challenge) protective immunity in all immunized mice.

Abstract: The invention provides, inter alia, methods for modulating DNA sensing pathways and the innate immune responses downstream of such pathways. The invention contemplates methods for down-regulating this pathway and/or the downstream innate immune response. The invention further contemplates methods for up-regulating this pathway and/or the downstream innate immune response.

Abstract: The usage of a stem cell in preparation of a tooth-like structure is provided. And a culture medium, a method for preparing an epithelial-like cell, a kit for preparing an ameloblast, a method for preparing an ameloblast are also provided. Specifically, the culture medium comprises a basal medium, which is DMEM/F12 medium; N2 supplement; retinoic acid; and BMP-4.

Abstract: Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.

Abstract: This invention provides compositions for use in distributing active agents for treating a malignant tumor in a subject. The compositions contain RNAi molecules targeted to a human GST-?, along with RNAi molecules targeted to a human p21, and a pharmaceutically acceptable carrier. The carrier can include nanoparticles composed of an ionizable lipid, a structural lipid, one or more stabilizer lipids, and a lipid for reducing immunogenicity of the nanoparticles. This invention further provides methods for preventing or treating a malignant tumor by administering a therapeutically effective amount of an RNAi composition.

Abstract: The present invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Leber congenital amaurosis.

Abstract: The present invention provides nucleic acid inhibitors of MYH7B and compositions thereof. The present invention also provides methods of treating or preventing a cardiac disorder such as cardiac hypertrophy, myocardial infarction, or heart failure in a subject by administering to the subject an inhibitor of MYH7B. The present invention further provides methods of modulating the activity or expression of ?-MHC in cardiac cells of a subject by administering to the subject an inhibitor of MYH7B.

Abstract: Compounds useful as labels with properties comparable to known fluorescent compounds. The compounds are conjugated to proteins and nucleic acids for biological imaging and analysis. Synthesis of the compounds, formation and use of the conjugated compounds, and specific non-limiting examples of each are provided.

Abstract: A hyaluronic acid conjugates including hyaluronic acid, a disulfide bond-containing crosslinking agent, and a cationic, amphiphilic polymer; a hyaluronic acid-nucleic acid complex in which a nucleic acid is bound to the hyaluronic acid conjugate; a composition in which the hyaluronic acid-nucleic acid complexes are crosslinked with each other; a nucleic acid delivery composition including the hyaluronic acid-nucleic acid complex; and a method of nucleic acid delivery using the hyaluronic acid-nucleic acid complex.

Abstract: The present invention relates to antisense of oligonucleotides that modulate the expression of and/or function of Fibroblast growth factor 21 (FGF21), in particular, by targeting natural antisense polynucleotides of Fibroblast growth factor 21 (FGF21). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of FGF21.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of RNase H1, in particular, by targeting natural antisense polynucleotides of RNase H1. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of RNASE H1.

Abstract: Embodiments disclosed herein relate to methods and systems for analysis of melting temperatures, and particularly to analysis of duplex nucleic acids.

Abstract: Antimicrobial peptides represent a relatively new discovery in the immune system path-way. Recent designs of synthetically engineered antimicrobial peptides have demonstrated increased potency and efficacy/tolerability, enhanced specificity, and reduced toxicity in comparison. One such peptide, XYLENTRA, has shown/demonstrated significant promise from significant in vitro studies against a large number of pathogens. Additionally, extensive animal studies have shown that the XYLENTRA is an antimicrobial peptide against a large number of pathogens. The XYLENTRA is also solute resistant. The test compound Xylentra® has shown/demonstrated significant antibacterial activity on test organisms Staphylococcus aureus MTCC 96 and Pseudomonas aeroginosa. MTCC 741. A substantial decrease in the microbial population level was observed in animals treated with peptide using the protocol described in detail in the application.

Abstract: Provided are: a novel test method for rheumatoid arthritis; and a kit for rheumatoid arthritis test, which is used in the novel test method for rheumatoid arthritis. A test method for rheumatoid arthritis according to the present invention is characterized by comprising a step for measuring the amount of talin in the plasma or serum of an animal subject. This measurement is carried out, for example, by an immunological method using an antibody which binds to talin. A kit for rheumatoid arthritis test according to the present invention is used for such a test method and contains, for example, a solid-phase carrier to which an antibody that binds to talin is affixed.

Abstract: One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene in vivo. The dsRNA duplex comprises one or more xylo modifications in one or both strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for in vivo therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.

Abstract: This invention relates generally to methods and compositions for diagnosing and treating disorders associated with elevated levels of Toll-like Receptor 4 (TLR4) ligands and other biomarkers. The invention also relates to methods of treating, delaying the progression of, or otherwise ameliorating a symptom of a disorder associated with elevated levels of TLR4 ligands and other biomarkers using agents that interfere with or otherwise antagonize TLR4 signaling, including neutralizing anti-TLR4 antibodies.

Abstract: The invention provides polymer compositions for cell and drug delivery.

Abstract: The present invention relates to pluripotent stem cells, particularly to pluripotent embryonic-like stem cells. The invention further relates to methods of purifying pluripotent embryonic-like stem cells and to compositions, cultures and clones thereof. The present invention also relates to a method of transplanting the pluripotent stem cells of the present invention in a mammalian host, such as human, comprising introducing the stem cells, into the host. The invention further relates to methods of in vivo administration of a protein or gene of interest comprising transfecting a pluripotent stem cell with a construct comprising DNA which encodes a protein of interest and then introducing the stem cell into the host where the protein or gene of interest is expressed. The present also relates to methods of producing mesodermal, endodermal or ectodermal lineage-committed cells by culturing or transplantation of the pluripotent stem cells of the present invention.

Abstract: Methods and compositions involving miR-122, miR-15b, miR-21, and miR-155, which are useful for the treatment of various diseases, such as cancers, are described. Further described are methods and compositions useful for increasing, activating, or regulating NK cells and surface antigens.

Abstract: The invention relates to methods for differentiating progenitor cells into insulin producing pancreatic islet cells and compositions and methods for using such cells.

Abstract: The present invention relates to methods of identifying oligonucleotides capable of modulating the immune system in a mammalian subject, comprising analysis of which tertiary structural type said oligonucleotide adopts, in phosphate-buffered saline solution. Further, the invention provides oligonucleotides identifiable by the methods of the invention and to their use in methods of treating diseases, such as inflammatory diseases, autoimmune diseases, infectious diseases, neurodegenerative diseases and cancer.

Abstract: Methods and agents for reducing a level of an acetylated Tau polypeptide in a cell are provided. Methods for treating a tauopathy in an individual are also provided. Also provided is a method for diagnosing a cognitive impairment disorder in an individual. Methods for identifying an agent suitable for treating a tauopathy are also provided.

Abstract: Oligonucleotide conjugates, where an oligonucleotide is covalently attached to an aromatic system, are provided. In particular embodiments the oligonucleotide is complementary to the RNA component of human telomerase and is covalently attached to a nucleobase via an optional linker. The conjugates inhibit telomerase enzyme activity.

Abstract: A method of culturing mesenchymal stem cells (MSCs) is provided. The method comprising culturing a population of the MSCs in a medium comprising an aryl hydrocarbon receptor antagonist, thereby culturing MSCs.