Abstract: The present invention provides for novel compositions and methods for delivering genes of interest to stem cells using vectors that contain differentiation-specific transcriptional regulatory elements. For example, stem cells in the internal epithelia could be transfected with a vaccine construct, which has an epithelial cell differentiation-specific promoter driving the expression of viral envelope proteins. When the promoter used is specific for terminally differentiated epithelial cells, then the viral envelope proteins will be expressed only in the upper part of the epithelia and therefore, stimulate the immune response. The infected epithelial stem cells in the basal layer will continue to produce new antigen-expressing cells, without being eliminated by the immune response. This invention will be useful in the development of vaccines against viral agents that target the internal mucosa like HIV.

Abstract: Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.

Abstract: The present invention provides a method of expressing at least one heterologous nucleic acid sequence in a cell, the method comprising introducing at least one heterologous nucleic acid sequence into a cell by infecting said cell with a recombinant negative-strand RNA virus vector comprising said at least one heterologous nucleic acid sequence, wherein the recombinant negative-strand RNA virus vector includes a viral genome coding for a mutated P protein, which leads to a loss of the viral genome replication ability without a loss of the viral transcription ability, and wherein said at least one heterologous nucleic acid sequence encodes a cellular reprogramming or programming factor or a therapeutic protein. In addition, the present invention provides a cell or a population of cells prepared in vitro by said method as well as a pharmaceutical composition comprising said cell or population of cells.

Abstract: The present disclosure provides, inter alia, genetically encoded recombinant peptide biosensors comprising analyte-binding framework portions and signaling portions, wherein the signaling portions are present within the framework portions at sites or amino acid positions that undergo a conformational change upon interaction of the framework portion with an analyte.

Abstract: The present invention relates to a polypeptide that is capable of inhibiting transcription and expression of fatty acid synthase (FAS) and the polynucleotides encoding therefor, as well as the use thereof. Specifically, the present invention relates to a polypeptide that can inhibit the transcription and expression of FAS at the molecular level, the cellular level and in vivo, and can therefore prevent the overexpression of FAS. Said polypeptide and related peptidomimetics, including functional fragments or functional varieties thereof, and the genes encoding therefor, can be widely used in preventing and treating tumors such as liver cancer, and diseases closely related to the metabolism of fatty acid synthase, such as fatty liver and obesity.

Abstract: The present invention relates to Huwentoxin-IV variants, polynucleotides encoding them, methods of making and using the foregoing, and methods of alleviating pain with peptide inhibitors of Nav1.7.

Abstract: Virus-like particles (VLPs) of mammalian-hosted viruses, such as SARS-CoV and influenza viruses, have been recombinantly produced from Vero cells. The VLPs closely emulate the exterior of authentic virus particles and are highly immunogenic. They can elicit not only humoral but also cellular immune responses in a mammal. Compositions and methods related to the VLPs are also described.

Abstract: Analyte sensors, methods for producing and using analyte sensors, methods of detecting and/or measuring analyte activity, detecting pH change, and/or, controlling the concentration of an analyte in a system, are disclosed. Embodiments of the analyte sensors according to the disclosure can provide an accurate and convenient method for characterizing analyte activity, detecting pH change, controlling the concentration of an analyte in a system, and the like, in both in vivo and in vitro environments, in particular in living cell imaging.

Abstract: The present invention relates to polypeptides and their uses as apelin inhibitors. More particularly, the present invention relates to a polypeptide comprising the sequence as set forth in SEQ ID NO:1 wherein at least one arginine residue at position 18, 19, 22 or 23 has been substituted or deleted.

Abstract: The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.

Abstract: The present invention relates generally to the use of recombinant adeno-associated viruses (rAAV) for gene delivery and more specifically to the use of rAAV to deliver genes encoding human immunodeficiency virus entry inhibitors to target cells in mammals.

Abstract: The present invention discloses a cell system, as a host cell to be infected with an F gene-deficient virus, which can constitutively and stably express the F protein, and a method for producing an F gene-deficient virus by utilizing the cell. A non-proliferative human parainfluenza type 2 virus vector is produced by co-culturing an F gene-deficient human parainfluenza type 2 virus with a Vero cell having the F gene of human parainfluenza type 2 virus in such a manner that the F gene is non-inducibly expressed, and isolating viral particles from a culture supernatant.

Abstract: Provided herein are chimeric influenza hemagglutinin (HA) polypeptides, compositions comprising the same, vaccines comprising the same, and methods of their use.

Abstract: The present invention discloses a cell system, as a host cell to be infected with an F gene-deficient virus, which can constitutively and stably express the F protein, and a method for producing an F gene-deficient virus by utilizing the cell. A non-proliferative human parainfluenza type 2 virus vector is produced by co-culturing an F gene-deficient human parainfluenza type 2 virus with a Vero cell having the F gene of human parainfluenza type 2 virus in such a manner that the F gene is non-inducibly expressed, and isolating viral particles from a culture supernatant.

Abstract: The present invention is directed to novel tissue protective peptides. The tissue protective peptides of the invention may bind to a tissue protective receptor complex. In particular, the present invention is drawn to tissue protective peptides derived from or sharing consensus sequences with portions of cytokine receptor ligands, including Erythropoietin (EPO), that are not involved in the binding of the ligand to the receptor complex, e.g., to the EPO receptor homodimer. Accordingly, the tissue protective peptides of the invention are derived from the amino acid sequences of regions of cytokine receptor ligands that are generally located on or within the region of the ligand protein that is opposite of the receptor complex, i.e., are generally derived from amino acid sequences of regions of the ligand protein that face away from the receptor complex while the ligand is bound to the receptor.

Abstract: The present invention relates to a cell line in which an expression construct is introduced into a genomic DNA, the expression construct including: (a) a promoter operable in animal cells and heterologous to adenoviruses; and (b) a modified adenovirus E1 coding gene sequence of SEQ ID NO:1 operatively linked to the promoter. According to the present invention, the cell line of the present invention is a novel cell line which is less likely to produce a replication competent adenovirus (RCA). The adenovirus producing cell line of the present invention has a low possibility of producing RCA due to homologous recombination, when compared with conventional cell lines. Therefore, this makes it possible to regulate the required amount of virus during gene therapy using the adenovirus and prevent tissue damage and toxic effects caused by overproduction of the adenovirus.

Abstract: The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system.

Abstract: The present invention relates to new methods to promote sialylation of glycoconjugates, including recombinant glycoproteins, in glycoconjugate production systems. The invention relates to methods to promote efficient glycoconjugate sialylation in recombinant expression systems, by providing simpler and more economical ways to produce large intracellular pools of sialic acid precursors. The invention is directed to nucleic acids, vectors, and cells harboring vectors comprising nucleic acids encoding enzymes involved in the synthesis of sialic acid precursors, and cells harboring these nucleic acids in combination with nucleic acids encoding glycosyltransferases, including sialyltransferases, to facilitate the production of humanized recombinant glycoproteins in bacterial, fungal, plant, and animal cell expression systems.

Abstract: A method of cultivating cells in a bag is disclosed, which comprises the steps of: a) providing a sterile flexible bag comprising a filter material which is attached to a wall of the culture bag, wherein the filter material delimits a chamber inside the bag and wherein the chamber is fluidically connected to a first port in a wall of the bag, b) providing agitation means for the bag, c) introducing cell culture medium, microcarriers and cells to the bag, d) cultivating cells in the bag with agitation provided by the agitation means, allowing formation of a suspension of microcarriers with immobilized cells and supplying at least one gas via a second port (not shown) in a wall of the bag, e) removing cell debris and/or free cells together with liquid from said suspension of microcarriers through the filter material and the first port.

Abstract: Described herein are isolated polypeptides each containing one or more receptor-binding sites of toxin A (tcdA) of Clostridium difficile (Cd), nucleic acids encoding the polypeptides, and methods of using the polypeptides and nucleic acids.

Abstract: The current disclosure provides a method for the creation of a high-density cryopreserved cell bank using perfusion culture techniques and non-centrifugal concentration of cells. Methods of production using this high-density cryopreserved cell bank are also provided.

Abstract: Disclosed herein are methods and compositions for inactivating a FUT8 gene, using fusion proteins comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding said fusion proteins are also provided, as are cells comprising said polynucleotides and fusion proteins.

Abstract: Methyl-lysine affinity reagents created by engineering the 3×MBT methyl-lysine binding domain repeat of lethal (3) malignant brain tumor-like protein 1 (L3MBTL1) are disclosed. In particular, the invention relates to affinity reagents and affinity chromatography media comprising the 3×MBT domain repeat and methods of using such affinity reagents in detection, purification, and proteomic profiling of methylated proteins and peptides.

Abstract: Compounds are provided having inter alia good duration of action, high potency and/or convenient dosing regimens including once weekly administration. The compounds are engineered polypeptides which incorporate an albumin binding domain in combination with one or more biologically active polypeptides. Also provided are pharmaceutical compositions and methods of treatment for diseases and disorders including lipodystrophy, dyslipidemia, hyperlipidemia, overweight, obesity, hypothalamic amenorrhea, Alzheimer's disease, leptin deficiency, fatty liver disease or diabetes (including type I and type II). Additional diseases and disorders which can be treated by the compounds and methods described herein include nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver disease (NAFLD), metabolic syndrome X and Huntington's Disease.

Abstract: The present invention provides a chimaeric coronavirus S protein which is based on an S protein from a coronavirus strain with restricted tissue tropism, but which comprises at least part of the S2 subunit from a coronavirus strain with extended tissue tropism, such that a virus comprising the chimaeric S protein has extended tissue tropism. The present invention also provides a virus comprising such a chimaeric S protein.

Abstract: The document provides nucleic acids, polypeptides, and viruses containing nucleic acids and/or polypeptides. The document also provides methods for using viruses to treat cancer patients. Specifically, the document provides nucleic acid molecules encoding viral hemagglutinin (H) polypeptides, viral H polypeptides, and viruses containing nucleic acids and/or H polypeptides. Such viruses are useful for vaccinations and for treating cancer patients as the viruses are not shed.

Abstract: Neuroinvasive and neurovirulent strain of the West Nile virus, named IS-98-ST1, nucleic acid molecules derived from its genome, proteins and peptides encoded by said nucleic acid molecules, and uses thereof.

Abstract: The present invention provides novel nucleotides sequences, protein sequences, immunogenic compositions, vaccines, and methods that relate to making and using new porcine parvovirus 5B (PPV5B) that infects, inter alia, domestic swine. The compositions and methods provide for the detection of infections by said new virus, monitoring genetic changes in the viral sequences in wild and domestic animals and herds, and making and using novel vaccines for protecting animals from infection by the virus.

Abstract: Compounds are provided having inter alia good duration of action, high potency and/or convenient dosing regimens including oral administration, and reduced immunogenicity. The compounds are engineered polypeptides which incorporate an albumin binding domain in combination with one or more biologically active polypeptides. Also provided are pharmaceutical compositions and methods of treatment for diseases and disorders including obesity and overweight, diabetes, dyslipidemia, hyperlipidemia, Alzheimer's disease, fatty liver disease, Short Bowel Syndrome, Parkinson's disease, and cardiovascular disease.

Abstract: Isolated nucleic acids encoding polypeptides that exhibit butyrylcholinesterase (BChE) enzyme activity are disclosed, along with molecular criteria for preparing such nucleic acids, including codon optimization. Methods of preparing modified and/or truncated BChE molecules having selected properties, especially selective formation of monomers, are also described. Vectors and cells containing and/or expressing the nucleic acids are also disclosed.

Abstract: The present invention relates generally to a method of modifying gene expression and to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention utilizes recombinant DNA technology to post-transcriptionally modify or modulate the expression of a target gene in a cell, tissue organ or whole organism, thereby producing novel phenotypes. Novel synthetic genes and genetic constructs which are capable of repressing delaying or otherwise reducing the expression of an endogenous gene or target gene in an organism when introduced thereto are also provided.

Abstract: The present invention provides variant activin IIB soluble receptor polypeptides and proteins capable of binding and inhibiting the activities of activin A, myostatin, or GDF-11. The present invention also provides polynucleotides, vectors and host cells capable of producing the variant polypeptides and proteins. Compositions and methods for treating muscle-wasting and other diseases and disorders are also provided.

Abstract: C-terminal endostatin polypeptides are disclosed herein. Polynucleotides encoding these polypeptide, host cells transformed with the polynucleotides, and methods of using these polypeptides and polynucleotides are disclosed. Uses of these polypeptide, polynucleotides and expression vectors include the treatment of fibrosis in a subject. Thus, methods are provided for treating fibrosis, including fibrosis of the skin and/or the lung.

Abstract: Described are methods for determining the permissiveness of a cell for a virus that is a member of the family Arteriviridae or Coronaviridae or Asfarviridae, in particular, for Porcine Reproductive and Respiratory Syndrome Virus (PRRSV). Further described are methods and compositions related to the generation of host cells permissive for a virus that is a member of the family Arteriviridae or Coronaviridae or Asfarviridae, in particular, for PRRSV. Methods of utilzing the cells thus identified or thus generated, in preparing a culture of a virus that is a member of the family Arteriviridae or Coronaviridae or Asfarviridae, as well as the use of the virus for the purpose of vaccine production or diagnosis, are also described.

Abstract: The invention provides novel polypeptides having at least one biological activity of cardiotrophin and improved biologic drug-like properties, and polynucleotides encoding the polypeptides of the invention. The polypeptides of the invention can be used therapeutically, such as, for example, in methods of tissue regeneration.

Abstract: Testosteronan, a heparosan analog having the structure [-4-D-GlcUA-?1,4-D-GlcNAc-?1-]n, is produced by testosteronan synthase, a single protein that is a dual-action catalyst that utilizes UDP-GlcUA and UDP-GlcNAc to synthesize a polysaccharide having the structure [-4-D-GlcUA-?1,4-D-GlcNAc-?1-]n.

Abstract: The invention provides novel Wnt polypeptides that have enhanced solubility and improved biologic drug-like properties, and polynucleotides encoding the Wnt polypeptides of the invention. The Wnt polypeptides of the invention can be used therapeutically, such as, for example, in methods of preventing or treating muscle loss and/or promoting muscle hypertrophy and growth.

Abstract: The present invention provides novel mammalian alpha-kinase proteins: melanoma alpha-kinase (MK), heart alpha-kinase (HK), kidney alpha-kinase (KK), skeletal muscle alpha-kinase (SK), and lymphocyte alpha-kinase (LK). In particular, a novel kinase type is herein provided, characterized by the presence of an alpha-kinase catalytic domain and an ion channel domain. Isolated nucleic acids of the alpha-kinases MK, HK, KK, SK and LK are provided. Methods for making the novel alpha-kinases, cells that express the alpha-kinases and methods for treating an animal in need of either increased or decreased activity of the alpha-kinases are provided.

Abstract: IL4/IL13-binding proteins comprise binding domains, which inhibit IL4/IL13 binding to IL4Ralpaha and common gamma chain complexes (Type 1) and inhibit IL4 binding to IL4Ralpha and IL13Ralpha1 complexes (Type 2), and IL13 binding to IL13Ralpha1 and/or IL13Ralpha2, are useful in the treatment of cancer, inflammatory, and other pathological conditions, such as allergic or fibrotic conditions, especially pulmonary conditions.

Abstract: It is disclosed herein that SPANX-B is uniquely expressed in a number of human tumors and that SPANX-B is an immunogenic antigen that is recognized by human T cells inducing helper CD4+ and cytolytic CD8+ T cell responses. Specific SPANX-B polypeptides and polynucleotides are disclosed that can be used to generate an immune response. In several embodiments, these polypeptides can be used for the treatment of a variety of cancers, including melanoma, colon carcinoma, ovarian cancer, breast cancer, myeloma, lung carcinoma and renal cancer.

Abstract: The present disclosure refers to a method for a specific, versatile and sensitive detection of IFN-/virus-induced genes, a method for quantifying IFN potency and activity in a pharmaceutical preparation or biological sample, a method for distinguishing between IFN- and viral induction, and/or for distinguishing between different viruses, and a method for the quantification of virus activity. Also, the invention provides the necessary molecular tools like expression active response constructs, suitable cell lines, an array to perform the method and a kit.

Abstract: The present invention provides vectors that contain and express in vivo the genes encoding VP2 and VP5 of African Horse Sickness Virus or an epitope thereof that elicits an immune response in a horse against African horse sickness virus, compositions comprising said vectors, methods of vaccination against African horse sickness virus, and kits for use with such methods and compositions.

Abstract: IL-17 Receptor binding proteins, including non-naturally occurring and recombinantly modified proteins, methods of making and using such molecules as therapeutic, prophylactic and diagnostic agents are provided.

Abstract: A novel approach for inhibiting FGF2/FGFR1-mediated signalling is presented which is based on FGFR1 mutations to introduce higher affinity for the natural GAG co-receptors into the soluble part of the FGF1 receptor, preferably into the D2/D3 domains.

Abstract: Embodiments are directed compositions related to Eilat virus and uses thereof.

Abstract: The present invention relates to cell culture methods and compositions that are essentially serum-free and comprise a basal salt nutrient solution and an ErbB3 ligand.

Abstract: A method for regenerating pancreatic tissue using recombinant periostin protein, a nucleic acid encoding said periostin and pharmaceutical compositions comprising said periostin are disclosed. Isolation of a nucleic acid encoding a periostin isoform, panc, is also taught.

Abstract: The invention relates to polypeptides and polynucleotides associated with trophoblast cell death, differentiation, invasion, and/or cell fusion and turnover, and uses of same in the prevention, diagnosis and treatment of conditions requiring regulation of trophoblast cell death, differentiation, invasion, and/or cell fusion and turnover. In particular aspects, diagnostic methods are disclosed for evaluating conditions such as preeclampsia utilizing matador polypeptides and polynucleotides encoding same.

Abstract: The present invention relates to a pol I promoter derived from Vero cells and a recombinant vector containing the same. When the pol I promoter derived from Vero cells according to the present invention is utilized, viruses can be manufactured efficiently, and consequently, the manufacture of both seasonal influenza vaccine and pandemic vaccine can be prepared more quickly to usefully address either situation.

Abstract: Von Willebrand's Factor (VWF) is produced using an expression vector that includes: 1) a DNA sequence encoding a functional VWF protein; and 2) regulatory DNA capable of effecting expression of that DNA sequence in a host cell transformed with the vector. Restriction fragment length polymorphisms (RFLP's) associated with the VWF gene are identified and used in a probe for determining the source of a VWF gene in a DNA sample. The gene for VWF is localized to the short arm of human chromosome 12 (12p).