Abstract: The present invention relates to a mutant human alpha-synuclein with increased toxicity compared to wild-type alpha-synuclein, or a homologue thereof, wherein the mutant alpha-synuclein or homologue thereof comprises at least one amino acid substitution selected from the group consisting of a substitution at the alanine at position 56 (A56), at the alanine at position 76 (A76), at the methionine at position 127 (M127) and/or at the valine at position 118 (V118), as defined in the claims. Further, the invention relates to a polynucleotide encoding the mutant alpha-synuclein or homologue thereof, or an expression vector comprising said polynucleotide, a cell comprising the polynucleotide or expression vector, as defined in the claims. Also, a non-human animal comprising the cell of the invention is provided, as defined in the claims. Finally, the invention provides methods for identifying a substance that prevents or reduces toxicity of alpha-synuclein, as defined in the claims.

Abstract: The present invention provides materials and methods to induce cell death by methuosis, a non-apoptotic cell death mechanism, to induce vacuolization without cell death, or to induce cell death without vacuolization. Small molecules herein are useful for treating cell proliferation disorders or anomalies, particularly, but not exclusively, cancer. Methods related to the research and pharmaceutical use of the small molecules are also provided herein.

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: The present invention relates to small RNAs, inhibitors thereof, inhibitors of enzymes producing thereof, and their use to modulate the response of a cell to a DNA damaging event. The invention concerns also a method to detect the presence or quantify DNA damage.

Abstract: Disclosed are methods for stimulating or increasing ?-cell replication or growth, e.g., for increasing insulin secretion, and methods of treating disorders such as diabetes, as well as related compositions and formulations.

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 present invention relates to a method for preparing stem cells in high concentration. The present invention makes it possible to grow stem cells in an amount sufficient to be clinically usable in a short time, and makes it possible to relatively efficiently enhance the ability of administered stem cells to efficaciously reach target tissue and exhibit an action in a stable fashion and can therefore dramatically increase the efficacy of cell therapy using stem cells.

Abstract: Process for detecting and identifying micropeptides (miPEPs) encoded by a nucleotide sequence contained in the sequence of the primary transcript of a microRNA and use thereof for modulating gene expression.

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 use of Akt3 as a biomarker for detecting the occurrence of epithelial-to-mesenchymal transition (EMT) in a subject, and the use of Akt3 inhibitors to treat cancer is disclosed herein. Also disclosed are various methods for detecting the occurrence of epithelial-to-mesenchymal transition (EMT) in a subject by measuring Akt3 expression and/or activity.

Abstract: The present invention relates to pluripotent stem cells obtained from the dental pulp of patients of different ages, the invention furthermore comprises cultures of said cells, means for obtaining them and pharmaceutical compositions comprising them, as well as their application in tissue regeneration.

Abstract: The present invention is directed to the use of mitotically and/or lethally inactivated stem cells for the repair of damaged organs and/or tissues. Stem cells are mitotically and/or lethally inactivated and transplanted into damaged tissue. Any form of ex vivo inactivation of stem cells may be used such that the stem cells cannot undergo mitosis or cell division before in vivo application. Mitotically and/or lethally inactivated stem may be used to ameliorate numerous disease, injury, traumatic, ischemic, aging, and/or degenerative conditions in different types of organs and/or tissues.

Abstract: The present invention provides an apparatus for culturing cells which may comprise two or more culture compartments and a pooling compartment, wherein each of said two or more culture compartments may be separated from the other culture compartments; each of said two or more culture compartments which may comprise a port for the addition or removal of medium; and the pooling compartment communicates with said two or more culture compartments. The present invention also relates to a stand for apparatus for culturing cells.

Abstract: We disclose a method comprising: (a) providing an embryonic stem (ES) cell; and (b) establishing a progenitor cell line from the embryonic stem cell; in which the progenitor cell line is selected based on its ability to self-renew. Preferably, the method selects against somatic cells based on their inability to self-renew. Preferably, the progenitor cell line is derived or established in the absence of co-culture, preferably in the absence of feeder cells, which preferably selects against embryonic stem cells. Optionally, the method comprises (d) deriving a differentiated cell from the progenitor cell line.

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: This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.

Abstract: Disclosed herein are methods and compositions for modulating activity of CXCR4 genes, for example using zinc finger transcription factors (ZF-TFs) or zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZF-TFs or ZFNs, vectors comprising polynucleotides encoding ZF-TFs or ZFNs and cells comprising polynucleotides encoding ZF-TFs or ZFNs and/or cells comprising ZF-TF or ZFNs are also provided.

Abstract: Novel hexon isolated from simian adenovirus serotype 19 encoded in the polynucleotide defined as SEQ ID NO: 3, hepervariable region thereof, chimeric adenovirus comprising the same, and therapeutic use thereof provides a solution to the problem of safety and effective systemic treatment for developing gene therapeutic agents using adenovirus.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a RRM2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a RRM2 gene using said pharmaceutical composition; and methods for inhibiting the expression of RRM2 in a cell.

Abstract: This disclosure relates to methods of producing induced pluripotent (iPS), multipotent, and/or lineage-committed stem cells from differentiated cells, maintaining iPS, multipotent, and/or lineage-committed cells in culture, and re-differentiating the iPS and multipotent stem cells into any desired lineage-committed cell type.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a cell.

Abstract: Disclosed are methods for determining efficacy of a cyclodextrin therapy in a subject afflicted with a disorder involving oxysterol accumulation. These methods comprise: obtaining a first body fluid sample from the subject prior to cyclodextrin administration; administering cyclodextrin; obtaining at least one second body fluid sample after the cyclodextrin administration; subjecting the body fluid samples to chromatography-mass spectroscopy analysis to determine concentration of 24-hydroxycholesterol and/or cholestane-3?,5?,6?-triol; and determining magnitude of difference between the 24-hydroxycholesterol and/or cholestane-3?,5?,6?-triol concentration of the body fluid samples, whereby an increase or stabilization of 24-hydroxycholesterol concentration, or a reduction of cholestane-3?,5?,6?-triol concentration in the at least one second sample compared to the first sample, indicates efficacy of the cyclodextrin therapy.

Abstract: The present invention relates to molecular approaches to the production of nucleic acid sequences, which comprises the genome of infectious hepatitis C virus. In particular, the invention provides nucleic acid sequences which comprise the genomes of infectious hepatitis C viruses of either genotype 3a (strain S52) or genotype 4a (strain ED43). The invention therefore relates to the use of the nucleic acid sequences and polypeptides encoded by all or part of the sequences in the development of vaccines and diagnostic assays for HCV and in the development of screening assays for the identification of antiviral agents for HCV. The invention therefore also relates to the use of viral particles derived from laboratory animals infected with S52 and ED43 viruses.

Abstract: A polypeptide containing an amino acid sequence having at least 60% identity to the amino acid sequence SEQ ID No. 1 or containing at least one amino acid fragment of at least 6 consecutive amino acid residues of the amino acid sequence SEQ ID No. 1 or having immunological cross-reactivity to the amino acid sequence SEQ ID No. 1 or fragments thereof, wherein the amino acid sequence SEQ ID No. 1 codes for an allergen and the polypeptide comprises at least one T cell epitope recognized by a T cell receptor specific for a molecule having the amino acid sequence SEQ ID No. 1.

Abstract: The present invention relates to methods of using meso-1,2,3,4-tetrahydroxybutane for the maintenance and/or improvement of biological cell function and activity, and for the prevention of improper cell functioning or cell death, in vitro, ex vivo, and in vivo over time and/or during exposure to stress. Meso-1,2,3,4-tetrahydroxybutane can be used to promote cell survival and as a cell protection agent, to increase cell viability, and to improve conversion of progenitor or stem cells to mature cells, whether in vitro, in vivo, ex-vivo, or transplanted.

Abstract: Methods and compositions for producing NKX6-1+ pancreatic progenitor cells and/or insulin producing cells from an endodermal cell population, the method comprising contacting the endodermal cell population with an EGF component, a Nicotinamide component and/or a Noggin component, optionally a combination of at least one EGF component and at least one nicotinamide component to induce the differentiation of at least one endodermal cell into a NKX6-1+ pancreatic progenitor cell, the combination optionally further comprising at least one Noggin component.

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

Abstract: Methods for culturing pluripotent stem cells on fiber scaffolds are provided which result in the expansion of the number of stem cells without loss of pluripotency. Cells obtained by such methods, implants containing such cells and medical methods using such cells are also disclosed.

Abstract: We disclose a method comprising: (a) providing an embryonic stem (ES) cell; and (b) establishing a progenitor cell line from the embryonic stem cell; in which the progenitor cell line is selected based on its ability to self-renew. Preferably, the method selects against somatic cells based on their inability to self-renew. Preferably, the progenitor cell line is derived or established in the absence of co-culture, preferably in the absence of feeder cells, which preferably selects against embryonic stem cells. Optionally, the method comprises (d) deriving a differentiated cell from the progenitor cell line.

Abstract: Provided are systems and methods for providing human cell cultures. Further provided are cultures of feeder cells for use in stem cell technology, as well as cultures, culture systems and methods for maintenance and propagating of stem cells in an undifferentiated state as well as for the development of somatic cells cultures from stem cells, the somatic cell cultures being free of extraembryonic cells.

Abstract: The present invention relates to the generation of anterior definitive endoderm (ADE) cells from embryonic stem cells and the differentiation of such cells to, for example, pancreatic or liver cells. The invention also relates to cell lines, cell culture methods, cells markers and the like and their potential uses in a variety of applications.

Abstract: The present invention relates to endodermal progenitor cells and methods of isolation, culture, differentiation and use thereof.

Abstract: The invention provides methods of preventing or treating diseases or disorders caused by biological agents or chemical agents in a subject (e.g., a mammal, such as a human) by administering genetically modified human umbilical cord perivascular cells.

Abstract: The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.

Abstract: Provided herein are novel methods and compositions utilizing adipose tissue-derived stromal stem cells for treating fistulae.

Abstract: Described herein is the identification of primate-specific glial cell line-derived neurotrophic factor opposite strand (GDNFOS) transcripts and encoded peptides. In particular embodiments, provided herein are three GDNFOS antisense transcripts, referred to as GDNFOS-1, GDNFOS-2 and GDNFOS-3. The GDNFOS-3 transcript encodes an ORF of 105 amino acids. Compositions comprising the GDNFOS transcripts and peptides are also provided by the present disclosure. Further provided are methods of treating a neurodegenerative or peripheral organ disease in a subject by administering a therapeutically effective amount of the disclosed GDNFOS nucleic acid molecules, peptides or compositions.

Abstract: This invention provides disc stem cells, processes for obtaining and culturing disc stein cells, and methods for repairing damaged or diseased disc tissue comprising the use of the disc stem cells of the invention.

Abstract: A biodegradable and biocompatible polyurethane composition synthesized by reacting isocyanate groups of at least one multifunctional isocyanate compound with at least one bioactive agent having at least one reactive group —X which is a hydroxyl group (—OH) or an amine group (—NH2). The polyurethane composition is biodegradable within a living organism to biocompatible degradation products including the bioactive agent. Preferably, the released bioactive agent affects at least one of biological activity or chemical activity in the host organism. A biodegradable polyurethane composition includes hard segments and soft segments. Each of the hard segments is preferably derived from a diurea diol or a diester diol and is preferably biodegradable into biomolecule degradation products or into biomolecule degradation products and a biocompatible diol. Another biodegradable polyurethane composition includes hard segments and soft segments.

Abstract: A reverse transcriptase encoded by L-1 (LINE-1) has been identified as a target molecule for treating or preventing cancers induced or mediated by this molecule. Method of treating or preventing such cancers in patients involves administration of a therapeutically effective amount of a composition having an inhibitor or antagonist of the reverse transcriptase in cells of the patients. The inhibitor or antagonist blocks lengthening of telomeres in telomerase negative cells. Methods and kits for detecting pathologically proliferating cells expressing L1RT are also disclosed.

Abstract: The use of interferon induced transmembrane protein 1, 2, or 3 (IFITM1, 2, or 3) as a viral restriction factor, and methods of using the same to produce virus, transgenic animals expressing exogenous IFITM1, 2, or 3, and methods of treating or inhibiting viral infections by targeting a gene identified herein.

Abstract: The present invention provides systems and methods for improving the efficiency of a transient gene delivery system to differentiating embryonic stem (ES) cells by serum starving the targeted cells for one to three days prior to transfection. Such a serum starvation surprisingly resulted in increased expression of a constitutively-controlled plasmid from 50.4% to 83.2% of the population and increased expression of a promoter/enhancer controlled plasmid from ˜1.4% to ˜3.7% of the population.

Abstract: The present invention relates to the production of gene sequences encoding chimerical membrane glycosyltransferases presenting an optimized glycosylation activity in cells transformed with the sequences, the gene sequences corresponding to the fusion: of a first nucleic acid coding for a C-terminal minimal fragment of the catalytic domain (CD) of the native full length glycosyltransferase, to a second nucleic acid coding for a transmembrane peptide comprising in its N-terminal region a cytoplasmic tail (CT) region located upstream from a transmembrane domain (TMD), itself located upstream of a stem region (SR), provided that at least one of these CT, TMD, SR peptides being different from the primary structure of the naturally occurring peptide counterparts present in the native glycosyltransferase from which is derived the CD fragment with optimal glycosyltransferase activity as defined above.

Abstract: Embodiments herein relate to compositions and methods for engraftment of and increasing survival of muscle cells in a subject in need thereof. In certain embodiments, compositions including myofibers and/or satellite stem cells may be administered to a subject. Other embodiments relate to compositions and methods for introducing one or more compounds to a subject using cell compositions disclosed herein. Still other embodiments relate to uses of these compositions in kits for portable applications and methods.

Abstract: A composition of a female germinal cell (egg) extract of pluricellular organisms in M-phase of the cell cycle, the extract being used for a mitotic remodeling of chromosomes of donor cells of pluricellular organisms, wherein the mitotic remodeling confers to the nucleus of the donor cells the ability to adapt themselves to the early embryonic development, in particular to the replication phases, in order to carry out the embryonic development or to obtain stem cells.

Abstract: Methods that may be used for the electrochemical detection of multiple parameters, including chemical compounds. Further provided are cells that may be used in the electrochemical detection of multiple parameters, including chemical compounds, as well as a kit for the electrochemical detection of multiple parameters, including chemical compounds.

Abstract: Aspects of the invention relate to novel methods and compositions for assessing the level of cellular uptake of a nanoparticle construct, assessing the level of target binding of a nanoparticle construct and assessing the levels of RNAs and proteins in a given cell.

Abstract: The present invention provides a method for cryoprotecting a biological specimen comprising the step of freezing said biological specimen in the presence of a hydrogel and in the absence of cryoprotectant.

Abstract: Oligonucleotide compounds modulate expression and/or function of Erythropoietin (EPO) polynucleotides and encoded products thereof. Methods for treating diseases associated with Erythropoietin (EPO) comprise administering one or more oligonucleotide compounds designed to inhibit the EPO natural antisense transcript to patients.

Abstract: A single-layer tissue sheet having a puncture strength of 2 kgf to 6 kgf. A valve, such as a heart valve, made of one or more leaflets formed from a single-layer tissue sheet. A method of making a tissue sheet having a puncture strength of 2 kgf to 5 kgf. The ultra-strong tissue sheets described herein have very long culture times, such as in excess of 20 weeks. Valves that comprise one or more leaflets made from the ultra-strong tissue sheets described herein may be delivered via trans-cathete aortic valve implantation.

Abstract: The present invention provides improved methods for producing RPE cells from human embryonic stem cells or from other human pluripotent stem cells. The invention also relates to human retinal pigmented epithelial cells derived from human embryonic stem cells or other human multipotent or pluripotent stem cells. hRPE cells derived from embryonic stem cells are molecularly distinct from adult and fetal-derived RPE cells, and are also distinct from embryonic stem cells. The hRPE cells described herein are useful for treating retinal degenerative diseases.