Abstract: A method for treating and/or diagnosing pain and the source or type of pain, shock, and/or inflammatory conditions in a subject. A method of using a therapeutically effective amount of a DNA or RNA aptamer that shows high affinity for OLAMs to at least partially treat pain, shock, and/or inflammatory conditions in a subject. The DNA or RNA aptamer that shows high affinity for OLAMs may be coupled to a plasma protein binding compound or a pharmacologically active agent. A method of treating and or diagnosing pain, shock, and/or inflammatory conditions in a subject may include inactivating or preventing at least one linoleic acid metabolite to treat certain conditions (e.g., pain, shock, and/or inflammation) using a DNA or RNA aptamer that shows high affinity for OLAMs.

Abstract: The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.

Abstract: Gene encoding human glucokinase mutant is provided. The gene has the nucleotide sequence chosen from the nucleotide sequence listed as SEQ ID NO:2 and the nucleotide sequence wherein the ORF region encodes the same amino acid sequence as the one encoded by ORF region (position 487 to 1884) of SEQ ID NO:2 and the rest of the region is same as the non-ORF region of SEQ ID NO:2. Human glucokinase mutant encoded by the gene, the recombinant vectors carrying the gene, the hosts comprising the vectors, pharmaceutical compositions thereof, uses thereof, and methods for treating and preventing diseases by using the same are provided. The human glucokinase mutant encoded by the gene has higher activity than that of the wild type human glucokinase, and thus provides a new way of controlling blood glucose and/or preventing and/or treating disturbance of carbohydrate metabolism, especially preventing and treating diabetes.

Abstract: This invention relates to the field of biotechnology or genetic engineering. Specifically, this invention relates to the field of gene expression. More specifically, this invention relates to novel substitution mutant receptors and their use in a Group H nuclear receptor-based inducible gene expression system and methods of modulating the expression of a gene in a host cell for applications such as gene therapy, large scale production of proteins and antibodies, cell-based high throughput screening assays, functional genomics and regulation of traits in transgenic organisms.

Abstract: Nucleic acid molecules from cannabis has been isolated and characterized and encode polypeptides having aromatic prenyltransferase activity. Expression or over-expression of the nucleic acids alters levels of cannabinoid compounds. The polypeptides may be used in vivo or in vitro to produce cannabinoid compounds.

Abstract: Disclosed herein are methods for controlling stem cell differentiation through the introduction of transgenes having Xic, Tsix, or Xite sequences to block differentiation and the removal of the transgenes to allow differentiation. Also disclosed are small RNA molecules and methods for using the small RNA molecules to control stem cell differentiation. Also disclosed are stem cells genetically modified by the introduction of Xic, Tsix, or Xite sequences.

Abstract: The invention pertains to a novel cell line, an HIV tat-rev dependent GFP-Gaussia luciferase Reporter cell line, known henceforth as the GGR cell line, that detects pseudotype and replication competent HIV (cloned or uncloned isolates, in cell media or human serum) rapidly and with high sensitivity. This GGR cell line provides an improved method of characterizing the entry phenotype of HIV envelope genes, and detecting and examining primary HIV samples in the context of laboratory research, clinical trial monitoring, and medical diagnostics. Examples include, but are not limited to, determining the functional HIV viral load, responsiveness to treatment, characterization of viral co-receptor usage (testing for viral co-receptor usage, i.e., CCR5 vs CXCR4, as required prior to prescribing FDA-approved CCR5 inhibitors), and characterization of other viral or drug resistance phenotypic properties to guide treatment.

Abstract: Methods, compositions and kits are provided for increasing the expression of a gene product in a cell by contacting the cell with a modified small activating RNA (saRNA) molecule, which provides for an increase in gene expression that is improved over the increase in expression provided by traditional saRNAs. These methods and compositions find use in any application in which an increase in gene expression in a cell is desired.

Abstract: Provided herein are methods for inhibiting expression of DOHH in a cell, and for inhibiting hypusination of eIF5A in a cell, the methods comprising contacting a cell with a miRNA or a nucleic acid molecule encoding the miRNA, wherein the miRNA binds to the 3?UTR of the DOHH mRNA and wherein binding results in a reduction in DOHH expression. Also provided are methods for reducing cellular proliferation and for treating diseases associated with abnormal cellular proliferation.

Abstract: This invention provides a system for producing differentiated cells from a stem cell population for use wherever a relatively homogenous cell population is desirable. The cells contain an effector gene under control of a transcriptional control element (such as the TERT promoter) that causes the gene to be expressed in relatively undifferentiated cells in the population. Expression of the effector gene results in depletion of undifferentiated cells, or expression of a marker that can be used to remove them later. Suitable effector sequences encode a toxin, a protein that induces apoptosis; a cell-surface antigen, or an enzyme (such as thymidine kinase) that converts a prodrug into a substance that is lethal to the cell. The differentiated cell populations produced according to this disclosure are suitable for use in tissue regeneration, and non-therapeutic applications such as drug screening.

Abstract: Provided is a preventive, progression inhibitor or remedy for a disease one of the causes of which is the activation of the P13K/AKT signaling pathway or vice versa. A phosphorylation-inhibiting and/or dephosphorylating agent, which has an effect of inhibiting the phosphorylation at least at one of the phosphorylation sites of PTEN protein selected from the group consisting of T382, T383 and S380 and/or an effect of dephosphorylating the same, is prepared. Alternatively, a phosphorylation-inhibiting or dephosphorylating agent for PTEN is screened by a method comprising a step for confirming an ability of a test substance to inhibit the phosphorylation at least at one of the phosphorylation sites of PTEN protein selected from the group consisting of T382, T383 and S380 or a dephosphorylation ability thereof. Then, a substance having an effect opposite to the inhibition of PTEN phosphorylation or dephosphorylation thereof, e.g.

Abstract: The invention is directed to a chimeric gammaretrovirus comprising an gammaretroviral virion which contains a lentiviral Vpx protein and methods of use thereof. In a particular aspect, the chimeric gammaretrovirus is a chimeric murine leukemia virus (MLV) comprising an MLV virion which contains a lentiviral Vpx protein. The invention is also directed to use of the chimeric gammaretrovirus to produce a MLV that can transduce a non-dividing cell (G1/S/G2), transduce a non-dividing cell; enhance the ability of a MLV to transduce a non-dividing cell; transduce a quiescent (G0) cell; and enhance the ability of a human immunodeficiency virus 1 (HIV-1) to transduce a quiescent (G0) cell.

Abstract: A method of altering a eukaryotic cell is provided including transfecting the eukaryotic cell with a nucleic acid encoding RNA complementary to genomic DNA of the eukaryotic cell, transfecting the eukaryotic cell with a nucleic acid encoding an enzyme that interacts with the RNA and cleaves the genomic DNA in a site specific manner, wherein the cell expresses the RNA and the enzyme, the RNA binds to complementary genomic DNA and the enzyme cleaves the genomic DNA in a site specific manner.

Abstract: The invention provides methods of treating diseases, disorders or injuries involving motor neuron survival and axonal growth, including amylotrophic lateral sclerosis, by the administration of a LINGO-2 antagonist. An exemplary method for promoting survival of a motor neuron, comprising contacting said motor neuron with an effective amount of a composition comprising a LINGO-2 antagonist selected from the group consisting of: (i) a soluble LINGO-2 polypeptide; (ii) a LINGO-2 antibody or antigen-binding fragment thereof; (iii) a LINGO-2 antagonist polynucleotide; (iv) a LINGO-2 aptamer; and (v) a combination of two or more of said LINGO-2 antagonists.

Abstract: The present invention related to a polynucleotide sequence and an expression vector comprising at least one gene encoding a stress resistance protein, at least one gene encoding a selection marker, at least one gene encoding an expression protein, at least one matrix attachment region and a transcription terminator, all of which are operably connected to each other. The present invention further relates to a host cell comprising the expression vector. The present invention also relates a method of producing a cell line.

Abstract: The disclosure relates to a method of reprogramming one or more somatic cells, e.g., partially differentiated or fully/terminally differentiated somatic cells, to a less differentiated state, e.g., a pluripotent or multipotent state. In further embodiments the invention also relates to reprogrammed somatic cells produced by methods of the invention, to uses of said cells, and to methods for identifying agents useful for reprogramming somatic cells.

Abstract: The present invention relates to agents and methods that are capable of augmenting NK-mediated killing of target cells by reducing inhibitory KIR signalling without reducing the binding of KIR to HLA-C. As described herein, transduction of negative signaling via KIR, upon binding of KIR to its HLA class I ligand, can involve a ligand-binding induced, conformational reorientation of the KIR molecules allowing interactions to form between adjacent KIRs in specific domains, leading to accelerated clustering. Methods and agents such as monoclonal antibodies for reducing KIR-mediated inhibition of NK cell cytotoxicity without reducing or blocking HLA-binding by, e.g., reducing or blocking dimerization of KIR, are provided.

Abstract: The present invention involves the use of transcription factors including Tbx5, Mef2C, Hand2, myocardin and Gata4 to reprogram cardiac fibroblasts into cardiomyocytes, both in vitro and in vivo. Such methods find particular use in the treatment of patients post-myocardial infarction to prevent or limit scarring and to promote myocardial repair.

Abstract: Regulatory elements, specifically insulators and transgene constructs containing insulator nucleic acid sequences, are disclosed herein. Methods of using insulators and transgene constructs including insulators to inhibit, delay, or prevent gene silencing are also disclosed herein.

Abstract: An oocyte and embryo-positioning apparatus enables a plurality of oocytes, embryos or other cell specimens to be held and positioned for injection therein of fluid molecular reagents such as DNA, RNA, protein, cells, spermatozoa and cells; and removal of cells, fragments of cells, cytoplasm, nuclei and cellular organelles. The oocytes and embryos are kept separate from each other in open marked or unmarked well-compartments that are located in the culture container. Each well-compartment comprises one or more finger like structures that can position one oocyte or one embryo. The lanes between the digits of the fingers may be sloped allowing the oocytes or embryo to position at the narrowest and lowest point of the compartment. The method and oocytes and embryo-positioning apparatus of this invention includes a culturing container, such as a Petrie dish, in which the oocytes and embryos are held for micromanipulation.

Abstract: The invention provides monoclonal antibody 6B8 and related antibodies. The 6B8 antibody binds to an epitope within residues 3-12 of IAPP. The antibodies of the invention are useful, for example, for treating disorders associated with IAPP accumulation, particularly accumulation of IAPP deposits. Such disorders include type 2 diabetes, metabolic syndrome, impaired insulin tolerance, impaired glucose tolerance, insulinomas, and related conditions.

Abstract: Embodiments of the present invention provide for the facile generation of a stable recombinant fusion polypeptides with intrinsic fluorescent properties. The recombinant antibodies may be suitable for qualitative and/or quantitative immunofluorescence analysis. Generally, the fluorescent polypeptides include a fluorescent domain comprising a C-terminus and an N-terminus; a first antibody domain covalently linked to the C-terminus; and a second antibody domain covalently linked to the N-terminus.

Abstract: There is provided peptides for inhibiting growth of cancer cells, the peptides comprising the amino acid sequence RxKxKxxxxR wherein K and R are respectively lysine and arginine amino acid residues, each x is independently an amino acid, and 5 wherein the peptide has 50% or less amino acid sequence identity with the amino acid sequence RSKAKNPLYR (SEQ ID. No. 2). Each x amino acid may independently be an amino acid residue selected from the group consisting of alanine (A), valine (V), leucine (L), isoleucine (I), threonine (T) and serine (S) amino acid residues. There is also provided chimeric proteins incorporating a peptide of the amino acid sequence 10 RxKxKxxxxR, nucleic acids encoding for the peptide, expression vectors including a nucleic acid encoding the peptide for expression of the peptide, and methods for use of the peptide for inhibiting growth of cancer cells.

Abstract: The disclosure provides methods for increasing genome stability of an embryonic stem (ES) cell or induced pluripotent stem (iPS) cell, increasing telomere length in an ES or iPS cell, or both, for example by contacting an ES or iPS cell with an agent that increases expression of Zscan4 in the cell. Methods for increasing genome stability or increasing telomere length in a population of ES or iPS cells are provided, for example by selecting Zscan4+ ES or iPS cells from the population of ES or iPS cells (which can include both Zscan4+ and Zscan4? ES or iPS cells). Therapeutic methods of using ES or iPS cells expressing Zscan4 are also provided. Further provided are methods of treating cancer by administering a Zscan4 polynucleotide or Zscan4 polypeptide. Also provided are methods of inducing differentiation of isolated ES or iPS cells into germ cells.

Abstract: The present invention relates to a cell, which has been genetically modified relative to its wild type, so that in comparison with its wild type it is able to produce more ?-aminocarboxylic acids, more ?-aminocarboxylic acid esters or more lactams derived from ?-aminocarboxylic acids, starting from carboxylic acids or carboxylic acid esters. Furthermore, the present invention relates to a method for the production of a genetically modified cell, the cells obtainable by this method, a method for the production of ?-aminocarboxylic acids, of ?-aminocarboxylic acid esters or of lactams derived from ?-aminocarboxylic acids, the ?-aminocarboxylic acids, ?-aminocarboxylic acid esters or lactams derived from ?-aminocarboxylic acids obtainable by this method, a method for the production of polyamides based on ?-aminocarboxylic acids or based on lactams and the polyamides obtainable by this method.

Abstract: The present invention relates to a method for preparing an expression vector encoding a tailored recombinase, which tailored recombinase is capable of recombining asymmetric target sequences within the long terminal repeat (LTR) of proviral DNA of a plurality of retrovirus strains inserted into the genome of a host cell, as well as to the obtained expression vector, cells transfected with this, expressed recombinase and pharmaceutical compositions comprising the expression vector, cells and/or recombinase. Pharmaceutical compositions are useful, e.g., in treatment and/or prevention of retrovirus infection. In particular, asymmetric target sequences present in a plurality of HIV strains are disclosed, as well as tailored recombinases capable of combining these sequences (Tre 3.0 and 4.0) and expression vectors encoding them.

Abstract: The invention relates to kinase ligands and polyligands. In particular, the invention relates to ligands, homopolyligands, and heteropolyligands that modulate AKT activity. The ligands, homopolyligands, and heteropolyligands are utilized as research tools or as therapeutics. The invention includes linkage of the ligands, homopolyligands, and heteropolyligands to a cellular localization signal, epitope tag and/or a reporter. The invention also includes polynucleotides encoding the ligands, homopolyligands, and heteropolyligands.

Abstract: A chemical tool and its use in genome surgery includes P2E2 constructs of in, order, a cell penetration component, a DNA binding component and a restriction endonuclease. The method for performing genome surgery includes: a) providing one or more recombinant of the P2E2 constructs; b) penetrating a cell with the recombinant P2E2 protein construct; c) forming a protein product in the cell by the processes of transcription and translation or by direct introduction of the P2E2 protein construct to the cell; d) attaching the protein product of the P2E2 construct to one or more targeted genomic sequences within the cell; and e) the endonuclease of the P2E2 construct cutting both strands of the genome at target locations.

Abstract: Provided herein are methods for preventing or treating a viral infection in a subject, wherein the viral infection is mediated by a virus comprising one or more viral RNA molecules translated by a ribosomal shunting mechanism or a non-IRES mediated mechanism. The methods comprise administering to a subject an agent that reduces ribosomal protein (Rps25) expression or function. Also provided are methods of inhibiting or promoting ribosomal shunting-mediated translation or non-IRES mediated translation. Also provided are methods of screening for an agent that inhibits or promotes ribosomal shunting-mediated translation or non-IRES mediated translation.

Abstract: In this invention, a novel protein interaction domain is provided along with several of its variants. This domain is involved in protein-protein interactions with the Bcl-2 family of proteins. It is named BLID (Bcl2 family of proteins Like Interaction Domain). Several BLID peptides that could be useful for discovery of drugs to help fight pathological states like cancer are presented.

Abstract: Provided are methods and compositions useful for producing and propagating stem cells from fibroblasts.

Abstract: The present invention relates to a method for selecting induced pluripotent stem (iPS) cells.

Abstract: To efficiently identify and select a clone from clones of induced pluripotent stem cells (iPS cell) having low tumor formation rate in vivo when allowed to differentiate and transplanted in a living body, iPS cells of the clones are induced to differentiate, undifferentiated cells among the cells after the induction of differentiation are detected, and a clone having the content of the undifferentiated cell below a control is selected.

Abstract: The invention provides a method for influencing the stability of an antibody producing cell, comprising directly or indirectly influencing the amount of BCL6 and/or Blimp 1 expression product within said antibody producing cell. Stable antibody producing cells and cell lines are also provided, as well as methods for producing antibodies using such cells and/or cell lines.

Abstract: Treatment of prostate cancer by regional and prolonged release of one or more nucleotide-based RNAi agents is provided.

Abstract: The present invention provides new compositions for transposase-mediated fragmenting and tagging DNA targets. The invention relates to the surprising discovery that use of manganese ions (Mn2+) in transposase reactions improves the transposase reaction. It also relates to the surprising discovery that Mg2+ ions can be used in a transposase reaction with wild-type and/or engineered transposases at levels much higher than previously thought. The invention provides for the use of naturally-occurring transposases in in vitro reactions, as well as improved schemes for cleaving, tagging, and amplifying target DNA.

Abstract: An epidermal growth factor receptor (EGFR)-homing vector comprising a double-stranded RNA (dsRNA) molecule with an EGFR-binding peptide or polypeptide, is disclosed for use in combination with immune cells for treatment of cancer overexpressing EGFR.

Abstract: Disclosed herein are donor molecules comprising single-stranded complementary regions flanking one or more sequences of interest. The donor molecules and/or compositions comprising these molecules can be used in methods for targeted integration of an exogenous sequence into a specified region of interest in the genome of a cell.

Abstract: Disclosed herein include embodiments related to addition, deletion, or modification of DNA, RNA, or protein in a subject. In an embodiment, the DNA, RNA, or protein is endogenous. In an embodiment, the DNA, RNA, or protein is exogenous. Further embodiments relate to computerized systems for assisting in the disclosed methods.

Abstract: The present invention provides recombinant proteins comprising the amino acid sequence of an intracellular segment of CD40 and an amino acid sequence mediating the association of the recombinant protein with the constant region of an immunoglobulin heavy chain. The recombinant proteins according to the present invention are useful for inducing clonal expansion of a B cell having a predetermined antigen-specificity without the need for T cell or CD40L mediated co-stimulation. Thus, the present invention provides tools for clonal expansion of B cells specific for an antigen of interest and the production of B cells secreting antibodies specific for an antigen of interest. The recombinant proteins of the present invention may also be used for generating fully human monoclonal antibodies with a predetermined antigen-specificity from the B cell repertoire of a human subject.

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 provides diacylhydrazine ligands and chiral diacylhydrazine ligands for use with ecdysone receptor-based inducible gene expression systems. Thus, the present invention is useful for applications such as gene therapy, large scale production of proteins and antibodies, cell-based screening assays, functional genomics, proteomics, metabolomics, and regulation of traits in transgenic organisms, where control of gene expression levels is desirable. An advantage of the present invention is that it provides a means to regulate gene expression and to tailor expression levels to suit the user's requirements.

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: Isolated human multipotent adult stem cell and isolated populations of cells that include human multipotent adult stem cells are disclosed. Human hair-follicle derived multipotent adult stem cells and methods of preparing isolated populations of cells that include human multipotent adult stem cells are disclosed. Isolated human hair-follicle derived multipotent adult stem cell that can differentiate in culture into a neuronal cell, a glial cell, a melanocyte cell, a muscle cell, an osteocyte, a chondrocyte, and a lymphocyte. Isolated human hair-follicle derived multipotent adult stem cell that can grow in cell culture in spheres are disclosed.

Abstract: Mixtures of cell types can be analyzed by having at least two signal markers, with at least one at three different levels to provide a barcode for each cell type. The mixture of cells may be subjected to a common candidate moiety and the effect of the moiety on the cells determined along with identification of the cell by the barcode. Conveniently, surface marker proteins and labeled antibodies can be used to create the barcode and the cells analyzed with flow cytometry.

Abstract: It is described in vitro methods for expanding, detecting or isolating rare populations of antigen specific memory T cells. It is also described an in vitro method for obtaining a genetically modified memory T cell population. Uses of cells so obtained are also disclosed.

Abstract: The present invention provides autonomous replication sequences (ARSs) isolated from Nannochloropsis that support the replication of episomal DNA molecules (EDMs) in eukaryotic cells. The ARSs and EDMs provided herein can be used for expressing genes in organisms including algae and heterokonts.

Abstract: Disclosed are exosomes that include a packaging protein and a cargo RNA in which the packaging protein binds specifically to the cargo RNA. The packaging protein is a fusion protein that includes an RNA-binding domain and an exosome-targeting domain. The cargo RNA includes an RNA-motif that the RNA-binding domain of the fusion protein binds specifically such that the cargo RNA is packaged in the lumen of the exosomes.

Abstract: The present invention provides a polypeptide having the formula: St-R1-S1-Q-S2-R2 wherein St is a stalk sequence which, when the polypeptide is expressed at the surface of a target cell, causes the R and Q epitopes to be projected from the cell surface; R1 and R2 are a Rituximab-binding epitopes each having the an amino acid sequence selected from the group consisting of SEQ ID No. 1, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15 and 16 or a variant thereof which retains Rituximab-binding activity; S1 and S2 are optional spacer sequences, which may be the same or different; and Q is a QBEnd1O-binding epitope having the amino acid sequence shown as SEQ ID No. 2 or a variant thereof which QBEnd1O-binding activity. The invention also provides a nucleic acid sequence encoding such a polypeptide and uses thereof in adoptive cell transfer.

Abstract: Provided is a method of inserting a target sequence into a genome of a cell through positive selection or negative selection based on whether a marker is expressed.