Abstract: Methods of treating, reducing the risk of developing, or delaying the onset of an inflammatory disease are disclosed. The methods involved providing a subject with or at risk of developing an inflammatory disease and administering to the subject an effective amount of a first therapeutic composition comprising miR-124. Further provided are methods of diagnosing a subject with or at risk of developing an inflammatory disease.

Abstract: The present invention provides new anti-?5?1 antibodies, compositions and kits comprising the antibodies, and methods of making and using the antibodies.

Abstract: Human somatic cells are reprogrammed to become induced pluripotent stem cells (iPS cells) by the introduction of a minicircle DNA vector. Cells of interest include adipose stem cells.

Abstract: The present invention provides novel amino-lipids, compositions comprising such amino-lipids and methods of producing them. In addition, lipid nanoparticles comprising the novel amino-lipids and a biologically active compound are provided, as well as methods of production and their use for intracellular drug delivery.

Abstract: Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).

Abstract: A malignant tumor cell suppressor protein (a) or (b): (a) a protein comprising an amino acid sequence represented by SEQ ID No. 1; or (b) a protein comprising an amino acid sequence represented by SEQ ID No. 1, wherein one or more amino acid are deleted, substituted or added in the amino acid sequence set forth in SEQ ID No. 1.

Abstract: Disclosed are methods and compositions for early diagnosis, monitoring and treatment of retinal dystrophy, age-related macular degeneration, Bardet-Biedel syndrome, Bassen-kornzweig syndrome, best disease, choroidema, gyrate atrophy, congenital amourosis, refsun syndrome, stargardt disease and Usher syndrome. In particular, the invention relates to a protein, termed “Rdcvf1,” that is differentially transcribed and expressed in subjects suffering from retinal dystrophies and the like, such as retinal dystrophy and age-related macular degeneration compared with nonsufferers, antibodies which recognize this protein, and methods for diagnosing such conditions.

Abstract: Disclosed herein are methods and compositions for inactivating TCR genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.

Abstract: A composition includes an isolated cell; at least one particle within said cell; and at least one active agent associated with the particle, wherein the active agent is capable of being released from the cell. A method includes administration of such a cell to a subject.

Abstract: The present invention relates to a recombinant protein for siRNA delivery, which allows the efficient intracellular and in vivo delivery of siRNA. More particularly, the present invention relates to a recombinant protein that allows a siRNA binding protein to be located in the interior cavity of a capsid protein of HBV (Hepatitis B virus), in which siRNAs of interest bind to the siRNA binding protein to be encapsulated within the capsid shell, thereby providing stability against the external attack such as nucleases and achieving the efficient intracellular and in vivo delivery of siRNA by its release into the cytosolic space after cell uptake.

Abstract: The present invention is directed generally to eukaryotic host cells comprising artificial endosymbionts and methods of introducing artificial endosymbionts into eukaryotic host cells. The invention provides artificial endosymbionts that introduce a phenotype to host cells that is maintained in daughter cells. The invention additionally provides eukaryotic host cells containing magnetotactic bacteria.

Abstract: The invention provides methods of reducing or decreasing a size of a tumor or eliminating a tumor or inhibiting, decreasing, or reducing neo-vascularization or angiogenesis in a tumor in a patient by administering an adenovirus comprising a nucleic acid construct comprising a FAS-chimera gene operably linked to an endothelial cell-specific promoter. Also provided is a homogeneous population of an adenovirus comprising a FAS-chimera gene operably linked to an endothelial cell-specific promoter and its uses thereof.

Abstract: Disclosed are RNA constructs which function to activate or inactivate a biological process, e.g., may be designed for attachment to a polypeptide coding region. Such RNA constructs modulate translation of a polypeptide from the coding region in response to the presence of a target polynucleotide in an expression environment. Such RNA constructs include a weakened stem-loop structure which, when bound to the target polynucleotide, assumes stem-loop secondary structure and associates with an RNA binding protein. Association with the RNA binding protein modulates translation of the polypeptide coding region. Such RNA constructs also have three-way junction joining regions 3? and 5? of the stem-loop structure.

Abstract: Reprogramming substances capable of substituting for Klf4, selected from the group consisting of members of the IRX family (e.g., IRX6), members of the GLIS family (e.g., GLIS1), members of the PTX family (e.g., PITX2), DMRTB1, and nucleic acids that encode the same, are provided. Also provided are a method of producing iPS cells, comprising the step of introducing into a somatic cell both one or more kinds of the above-described nuclear reprogramming substances and a substance capable of inducing iPS cells from a somatic cell when combined with Klf4. Still also provided are iPS cells comprising an extraneous nucleic acid that encodes any one of the above-described nuclear reprogramming substances, that can be obtained by the method, and a method of producing somatic cells by inducing the iPS cells to differentiate.

Abstract: The present invention relates to a novel hepatocyte-like cell progenitor and/or a novel hepatocyte-like cell derived via definitive endoderm from human blastocyst-derived stem (hBS) cells, to a method for the preparation of such cells and to the potential use of such cells in e.g. pharmaceutical drug discovery and development, toxicity testing, cell therapy and medical treatment. In particular is presented a definitive endoderm derived hepatocyte-like cell with important liver-expressed marker genes and important metabolizing enzymes, as well as drug transporters.

Abstract: This invention relates to a mammalian artificial chromosome vector, which retains a human chromosome 7 fragment comprising human cytochrome P450 genes and is transmittable to progeny, wherein the human chromosome 7 fragment retains a region of approximately 1 Mb±500 Kb in size comprising at least a human CYP3A gene cluster, which region is located between chromosome markers AC004922 and AC073842, and to a non-human mammalian animal retaining the vector.

Abstract: The present invention relates to a composition for improving the migration potential of a stem cell, a method for evaluating the migration potential of a stem cell and a method for screening an adjuvant of cell therapy improving the migration potential of a stem cell. The present invention may be effectively used for enhancing the efficacy of neurological disease-treatment by inducing therapeutic stem cells to migrate efficiently to the lesion site.

Abstract: An object of the present invention is to stimulate a T cell without using a peptide/MHC tetramer. In the present invention, the step of supplying an antigen peptide to a T cell having a T cell receptor (TCR) that can recognize the antigen peptide on cell surface to form a complex of a major histocompatibility complex (MHC) molecule on the cell surface of the T cell and the antigen peptide is used, and the T cell is stimulated through recognition by TCR of the antigen peptide as the MHC molecule-antigen peptide complex on the cell surface of the same T cell. Such a stimulating and activating method would be applicable to not only T cells, but also various cells. According to the present invention, an antigen-specific T cell can be identified without establishing any antigen-specific T cell strain, and without using such a reagent as MHC/peptide tetramer. That is, a cancer-specific T cell can be efficiently and conveniently identified.

Abstract: A method of generating neural stem cells or motor neurons is disclosed, the method comprising up-regulating a level of at least one exogenous miRNA and/or down-regulating at least one miRNA using an agent which hybridizes to the miRNA in mesenchymal stem cells (MSCs) or down-regulating Related to testis-specific, vespid and pathogenesis protein 1 (RTVP-1).

Abstract: A nucleic acid comprising a transcription regulation sequence whose transcription is induced by a trans-acting factor of a human immunodeficiency virus and a gene encoding a polypeptide having an endoribonuclease activity specific to single-stranded RNA, wherein the gene is located in such a position that the expression of the gene can be regulated by the transcription regulation sequence; a method for production of a cell showing an inhibited replication of a human immunodeficiency virus therein, the method comprising the step of introducing the nucleic acid into a cell; and a method for treatment or prevention of a human immunodeficiency virus infection.

Abstract: The present disclosure provides methods of inducing smooth muscle cell differentiation. The present disclosure provides genetically modified cells comprising exogenous miR-143 and/or miR-145 nucleic acids; and artificial tissues comprising the genetically modified cells. The present disclosure provides methods and compositions for reducing pathological angiogenesis. The present disclosure provides methods of inducing therapeutic angiogenesis. The present disclosure provides methods, compositions, and devices for inhibiting vascular smooth muscle cell proliferation.

Abstract: A method of generating a population of cells useful for treating a nerve disease or disorder in a subject, the method comprising up-regulating a level of at least one exogenous miRNA in mesenchymal stem cells (MSCs) and/or down-regulating a level of at least one miRNA using a polynucleotide agent that hybridizes to the miRNA, thereby generating the population of cells useful for treating the nerve disease or disorder. Isolated populations of cells with an astrocytic phenotype generated thereby and uses thereof are also provided.

Abstract: Provided are a culture medium of an adipose-derived stem cell, a method for preparing the same, and a composition for promoting hair growth, in which the composition includes the culture medium. The adipose-derived stem cell (ADSC-T) according to the present invention exhibits long lifespan, improved cell proliferation rate, and extended proliferation period, as compared with a primary adipose-derived stem cell (ADSC), and thus, the adipose-derived stem cell (ADSC-T) can be usefully used for the study about the adipose-derived stem cell and the mass production of the culture medium of the adipose-derived stem cell. In addition, according to the present invention, the culture medium of the adipose-derived stem cell (ADSC-T) that expresses a T antigen of SV40 exhibits excellent hair growth effectiveness and can be usefully used as a raw material for the hair loss prevention and hair growing agents.

Abstract: This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.

Abstract: The invention provides for compositions and methods for identifying and validating modulators of cell fate, such as such as maintenance, cell specification, cell determination, induction of stem cell fate, cell differentiation, cell dedifferentiation, and cell trans-differentiation. The invention relates to reporter nucleic acid constructs, host cells comprising such constructs, and methods using such cells and constructs. The invention relates to methods for making cells comprising one or more reporter nucleic acid constructs using fluorogenic oligonucleotides. The methods relate to high throughput screens.

Abstract: The present invention relates to a new class of cationic polymers that self-assemble with a pH-sensitive dissolution switch, and their uses to deliver molecules of interest to a cell. The present invention also relates to compositions comprising said cationic polymers non-covalently associated with a molecule of interest, in particular with a siRNA.

Abstract: Disclosed herein are methods and compositions for the use of marrow adherent stem cells and their descendents; e.g., bone marrow-derived neural regenerating cells; in the treatment of various neurodegenerative disorders. In certain embodiments, bone marrow-derived neural regenerating cells transplanted to sites of neural degeneration stimulate growth and/or survival of host neurons.

Abstract: This invention provides a method for reducing hypertropic scarring resulting from dermal wound healing in a subject in need which comprises administering to the subject an antisense oligonucleotide which inhibits expression of connective tissue growth factor (CTGF) in an amount effective to inhibit expression of CTGF and thereby reduce hypertrophic scarring.

Abstract: The present invention provides a drug capable of causing cancer cells to restore anticancer drug sensitivity in cases in which cancer has acquired resistance to an anticancer drug and inducing cell death in cancer cells. The present invention specifically provides a cancer cell death inducing agent comprising REIC/Dkk-3 DNA as an active ingredient and having effects of potentiating an anticancer drug for cancer cells having anticancer drug resistance.

Abstract: A robust and genetically stable cell culture system for Hepatitis C Virus (HCV) genotype 3a is provided. A genotype 3a/2a (S52/JFH1) recombinant containing the structural genes (Core, E1, E2), p7 and NS2 of strain S52 was constructed and characterized in Huh7.5 cells. S52/JFH1 and J6/JFH viruses passaged in cell culture had comparable growth kinetics and yielded similar peak HCV RNA titers and infectivity titers. Direct genome sequencing of cell culture derived S52/JFH1 viruses identified putative adaptive mutations in Core, E2, p7, NS3, and NS5A; clonal analysis revealed that all genomes analyzed exhibited different combinations of these mutations. Finally, viruses resulting from transfection with RNA transcripts of five S52/JFH1 recombinants containing these combinations of putative adaptive mutations performed as efficiently as J6/JFH viruses in Huh7.5 cells and were all genetically stable after viral passage.

Abstract: Disclosed herein are methods and compositions for modulating the expression of a HLA locus or for selectively deleting or manipulating a HLA locus or HLA regulator.

Abstract: The present invention relates to a nucleic acid aptamer molecule that includes a domain that binds to an estrogen receptor, molecular complexes that include the nucleic acid aptamer molecule and an estrogen receptor, and constructed DMA molecules and expression systems, as well as host cells, that the contain an RNA aptamer molecule of the invention. Use of these aptamers and encoding constructs to inhibiting estrogen receptor activity in a cell and to treat estrogen receptor-positive cancers is also described.

Abstract: Methods are described for the delivery of one or more small interfering RNAs (siRNAs) to a eukaryotic cell using a bacterium. Methods are also described for using this bacterium to regulate gene expression in eukaryotic cells using RNA interference, and methods for treating an inflammatory disease or disorder. The bacterium includes one or more siRNAs or one or more DNA molecules encoding one or more siRNAs. Vectors are also described for use with the bacteria of the invention for causing RNA interference in eukaryotic cells.

Abstract: Provided are a culture medium for preparing neural stem cell and use thereof, the culture medium for preparing neural stem cell comprising: a basic culture medium suitable for the growth of stem cell, and a cell signal pathway inhibitor selected from at least one of GSK inhibitor, MEK inhibitor, TGF-? inhibitor, ROCK inhibitor and BMP inhibitor.

Abstract: The present invention relates inter alia to improvements in the production of chimaeric antibodies in non-human transgenic vertebrates such as mice and rats bearing one or more chimaeric antibody transgenes. In particular, the invention provides for improved non-human vertebrates and cells in which VpreB has been species-matched with the variable region of the chimaeric antibodies. Also, embodiments also provide for species-matching of the entire surrogate light chain for efficient pairing with chimaeric heavy chains during B-cell development in vivo in a non-human transgenic vertebrate setting.

Abstract: The application relates to compositions and methods of regulating an immune response comprising inhibitors of TLR7 and/or TLR9, such as immunoregulatory polynucleotides and/or immunoregulatory compounds. The application also relates to compositions and methods for predicting and/or determining responsiveness of a disease to treatment comprising inhibitors of TLR7 and/or TLR9.

Abstract: The present invention relates to immortalized avian cell lines suitable for production of biologicals or viruses for vaccination. In particular, the cell lines are derived from primary cells which are transformed with at least two viral or cellular genes, one of which causes cell cycle progression whereas the other interferes with innate protective mechanisms of the cell induced by dysregulated replication. The invention moreover relates to the production of said immortalized cell lines and their use for producing biologicals or viruses for vaccination.

Abstract: The present invention relates to methods and means for making Vitamin K-dependent protein compositions which are devoid or substantially devoid of protein contaminants. In particular, methods and means useful for the reduction or elimination of protein contaminants also being Vitamin K-dependent proteins are described.

Abstract: The present disclosure relates to a method for preparing recombinant glycoproteins with high sialic acid content. More specifically, for UDP-GlcNAc 2-epimerase/ManNAc kinase (GNE/MNK) enzyme where point mutation was induced by substituting arginine at position 263 by leucine only or by further substituting arginine at position 266 by glutamine, epimerase activity is constantly maintained, and overexpressed cells thereof experience an increase in intracellular cytidine monophosphate (CMP)-sialic acid content, irrespective of CMP-sialic acid concentration.

Abstract: Disclosed herein is an isolated nucleic acid molecule comprising a first nucleic acid sequence 5?-ACCCTGCCGCCTGGACTCCGCCTGT-3? (SEQ ID NO: 22), or a functional variant thereof, operably linked to a second, heterologous nucleic acid sequence. The isolated nucleic acid molecule can be DNA (in an expression vector) and RNA (mRNA, shRNA, orncRNA). Also disclosed is a microvesicle comprising the nucleic acid molecule and a microvesicle preparation comprising the microvesicle. Also disclosed is an in vitro method of producing a microvesicle preparation enriched for a specific RNA sequence by transfecting cells with the nucleic acid sequence, and isolating microvesicles generated therefrom. Methods of delivering therapeutic RNA to a subject are also disclosed.

Abstract: Isolated polynucleotides comprising a CLDN5 mini-promoter are provided. The mini-promoter may be operably linked to an expressible sequence, e.g. reporter genes, genes encoding a polypeptide of interest, regulatory RNA sequences such as miRNA, siRNA, anti-sense RNA, etc., and the like. In some embodiments a cell comprising a stable integrant of an expression vector is provided, which may be integrated in the genome of the cell. The mini-promoter may also be provided in a vector, for example in combination with an expressible sequence. The polynucleotides find use in a method of expressing a sequence of interest, e.g. for identifying or labeling cells, monitoring or tracking the expression of cells, etc.

Abstract: Methods and compositions are provided for generating novel nucleic acid molecules through targeted spliceosome mediated RNA trans-splicing (SMaRT™) that result in expression of a splicing isoform or variant thereof. The methods and compositions are based upon pre-trans-splicing molecules (PTMs) designed to interact with a target pre-mRNA molecule and mediate a trans-splicing reaction generating a novel chimeric RNA molecule encoding a splicing isoform for the treatment of a variety of gene isoform induced diseases such as cancer.

Abstract: Cells and cell lines that are genetically modified to express the hCox-2 enzyme, which results in the upregulation of prostaglandin F2 alpha (PGF2a) on the cells have been obtained. Encapsulated cell therapy devices containing such cells or cell lines that are capable of delivering PGF2a, as well as methods of using these devices to deliver PGF2a to the eye and to treat ophthalmic disorders in patients suffering therefrom are also described.

Abstract: The invention provides an isolated genetically modified non-mammalian organism, wherein the activity of acyl-CoA:sterol acyltransferase/sterol O-acyltransferase (EC 2.3.1.26) and/or diacylglycerol acyltransferase/diacylglycerol O-acyltranferase (EC 2.3.1.20) and/or lecithin cholesterol acyl transferase/phospholipid: diacylglycerol acyltransferase (EC 2.3.1.158) and/or acyl CoA-wax alcohol acyltransferase (EC 2.3.1.75) is reduced or abolished in comparison with a corresponding wildtype organism, methods of use of such an organism, shuttle vehicles for making such an organism and methods for producing such an organism.

Abstract: DCAMKL-1 has been identified as a biomarker for stem cells, as well as cancer stem cells. Methods of detecting the presence of at least one stem cell, methods of isolating stem cells, and methods of inhibiting growth of cancer cells utilizing DCAMKL-1 are disclosed herein.

Abstract: The present invention provides compositions comprising polytheylyene-dialkyloxypropyl conjugates (PEG-DAA), liposomes, SNALP, and SPLP comprising such compositions, and methods of using such compositions, liposomes, SNALP, and SPLP.

Abstract: Disclosed are methods and pharmaceutical compositions for inducing pancreatic hormone production.

Abstract: The present invention relates to methods for developing engineered T-cells for immunotherapy that are non-alloreactive. The present invention relates to methods for modifying T-cells by inactivating both genes encoding T-cell receptor and an immune checkpoint gene to unleash the potential of the immune response. This method involves the use of specific rare cutting endonucleases, in particular TALE-nucleases (TAL effector endonuclease) and polynucleotides encoding such polypeptides, to precisely target a selection of key genes in T-cells, which are available from donors or from culture of primary cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections.

Abstract: A method of using vaults as carrier molecules to deliver one or more than one substance to an organism, or to a specific tissue or to specific cells, or to an environmental medium. A vault-like particle. A method of preventing damage by one or more than one substance to an organism, to a specific tissue, to specific cells, or to an environmental medium, by sequestering the one or more than one substance within a vault-like particle. A method of delivering one or more than one substance or a sensor to an organism, to a specific tissue, to specific cells, or to an environmental medium. According to another embodiment of the present invention, there is provided a method of making vault-like particles, and making vault-like particles comprising one or more than one substance, or one or more than one sensor.

Abstract: Methods using somatic hypermutation (SHM) for producing polypeptide and nucleic acid variants, and nucleic acids encoding such polypeptide variants are disclosed. Such variants may have desired properties. Also disclosed are novel polypeptides, such as improved fluorescent proteins, produced by the novel methods, and nucleic acids, vectors, and host cells comprising such vectors.