Abstract: Disclosed are methods for detecting remnant cancer cells in a tissue sample.

Abstract: Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.

Abstract: This invention generally relates to a composition and method of using recombinant microRNAs (miRNA) and their hairpin-like precursors (pre-miRNA) as therapeutic drugs for treating Alzheimer's diseases (AD). More specifically, the present invention relates to the use of man-made miRNA miR-302 precursors (pre-miR-302) for AD therapy in humans. These pre-miR-302 molecules can be mass produced in prokaryotes as a form of DNA expression-competent DNA vectors and/or hairpin-like RNAs. As prokaryotic cells do not transcribe or process hairpin-like RNAs, the present invention also teaches a method for expressing pre-miRNAs in prokaryotes, i.e. pro-miRNA, using a novel hairpin-like RNA transcription mechanism newly found in prokaryotes.

Abstract: Inhibitory nucleic acids, e.g., antisense oligonucleotides (ASO) against PAR-TERRA RNA or other chromosome-specific TERRA transcripts (i.e., inclusive of chromosome-specific subtelomeric sequences), and methods of use thereof to downregulate expression of escapee genes on the inactive X chromosome, expression from the active X chromosome, subtelomeric autosomal loci (e.g., FSHD locus), or expression of autosomal genes involved in growth control and apoptosis, e.g., in cells and subjects with supernumerary X chromosomes and/or cancer and other human diseases.

Abstract: Briefly stated, the present invention provides compositions and methods for treating or preventing joint injuries utilizing hair follicle derived Non-Bulbar Dermal Sheath (“NDBS”) cells. Within one aspect of the invention methods are provided for isolating NBDS cells, comprising the steps of: (a) preparing vital hair; (b) cleaving the hair prepared in step (a) to remove the hair follicle bulb; (c) isolating Non-Bulbar Dermal Sheath tissue; and (d) cultivating the isolated Non-Bulbar Dermal sheath tissue to produce NBDS cells. Within one embodiment of the invention the vital hair is obtained by full skin biopsy from the occipital scalp of a subject.

Abstract: The present invention relates to the production of avian induced pluripotent stem cells from non-pluripotent somatic cells, including embryonic fibroblasts and adult somatic cells. In this method, avian (including quail or chicken) somatic cells are reprogrammed into a state closely resembling embryonic stem cells including the expression of key stem cell markers alkaline phosphatase, etc. by transfecting/transducing the non-stem cells with genes (preferably using a non-integrating vector as otherwise described herein or alternatively an integrating vector, such a lentiviral vector, retroviral vector or inducible lentiviral vector, among others) which express at least nanog, Lin28 and cMyc. In preferred aspects of the invention, the transfected/transduced vectors express nanog, Lig28, cMyc, Oct 4 (POU5F1 or PouV), SOX2 and KLF4. The induced stem cells which are produced contribute to all 3 germ layers, the trophectoderm and in certain aspects, the gonad in chimeric offspring.

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the ?-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

Abstract: Provided are a culture vessel with which an embryonic body can be formed efficiently from human embryonic stem cells, and a method for culturing human embryonic stem cells using the vessel. There is provided a vessel for culturing human embryonic stem cells, the culture vessel having two or more wells (1), wherein each of the wells (1) has a tubular body (2) and a funnel-shaped bottom (3) provided at one end of the body (2), the bottom (3) being a concave curved surface at the center (4) of the bottom (3) and the bottom (3) having an opening angle (?) in range of 60 to 100°. There is provided a method for culturing human embryonic stem cells by using said vessel for culturing human embryonic stem cells.

Abstract: Devices and methods for treating an abdominal incision or hernia are described. An implant for the restoration or prophylactic treatment of an abdominal wall comprises an elongate element and at least one sheet connected to the elongate element along a longitudinal axis of the elongate element. The elongate element is positioned along the line of incision, and the at least one sheet is secured to the abdominal muscles surrounding the incision.

Abstract: The invention provides biomarker profiles of metabolites and methods for screening chemical compounds including pharmaceutical agents, lead and candidate drug compounds and other chemicals using human stem-like cells (hSLCs) or lineage-specific cells produced therefrom. The inventive methods are useful for testing toxicity, particularly developmental toxicity and detecting teratogenic effects of such chemical compounds. Specifically, a more predictive developmental toxicity model, based on an in vitro method that utilizes both hSLCs and metabolomics to discover biomarkers of developmental toxicity is disclosed.

Abstract: This disclosure describes, generally, a modified form of CD 16, genetically-modified cells that express the modified CD 16, and methods that involve the genetically-modified cells. The modified form of CD 16 can exhibit increased anti-tumor and/or anti- viral activity due, at least in part, to reduced susceptibility to ADAM17-mediated shedding upon NK cell stimulation.

Abstract: The present specification discloses methods for detecting extremely low amounts of botulinum neurotoxin serotype A in samples, including complex matrices like blood, plasma, and serum.

Abstract: An object of the present invention is to provide a method for producing a mesenchymal stromal cell composition, comprising conveniently and aseptically separating high-purity amnion-derived MSCs by performing enzyme treatment only once. According to the present invention, the following are provided: a method for producing a mesenchymal stromal cell composition, comprising: performing enzyme treatment of an amnion with collagenase and thermolysin and/or dispase; and filtering the enzyme-treated amnion through a mesh; a method for producing a cryopreserved mesenchymal stromal cell composition; and a therapeutic agent comprising as an active ingredient the mesenchymal stromal cell composition for a disease selected from graft-versus-host disease, inflammatory bowel disease, systemic lupus erythematosus, liver cirrhosis, or radiation enteritis.

Abstract: The present invention relates to a magnetic particle separating device, and a method of separating and purifying nucleic acid or protein using the same. The device comprises: induction magnets (100); an induction magnet fixing part (200) having induction magnet fixing holes (210) for fixing the induction magnets (100); and a body (300) in which entry holes (310) are formed into which tubes (T) are inserted. Thus, the application and removal of a magnetic field to and from the body is made very convenient, so that the device can be very advantageously used in the separation and purification of nucleic acid or protein.

Abstract: The first method to cause a culture of human and other primate stem cells to directly and uniformly differentiate into a committed cell lineage is disclosed. Treatment of primate stem cells with a single protein trophoblast induction factor causes the cells to transform into human trophoblast cells, the precursor cells of the placenta. Several protein factors including bone morphogenic protein 4 (BMP4), BMP2, BMP7, and growth and differentiation factor 5 can serve as trophoblast-inducting factors.

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

Abstract: A nucleic acid containing a dopamine receptor type 2-specific promoter (D2SP) is provided. In certain embodiments, the nucleic acid includes a dopamine receptor type 2-specific promoter (D2SP), wherein the D2SP does not include exon 1 of a D2 receptor gene, wherein the D2SP comprises a Kozak sequence, and wherein the D2SP includes a nucleotide sequence having at least 95% sequence identity to the nucleotide sequence set forth in SEQ ID NO: 1. Also provided are expression vectors, genetically modified host cells and kits that include the subject nucleic acid.

Abstract: Compositions and methods for reducing or overcoming acquired resistance to cetuximab are provided. Compositions and methods for treatment of cetuximab-resistant cancers are also provided. In addition, methods of inhibiting the growth of EGFR-expressing tumor cells that are resistant to cetuximab therapy are provided herein.

Abstract: In some embodiments, compositions and methods relating to isolated artificial antigen presenting cells (aAPCs) are disclosed, including aAPCs comprising a myeloid cell transduced with one or more viral vectors, such as a MOLM-14 or a EM-3 myeloid cell, wherein the myeloid cell endogenously expresses HLA-AB/C, ICOS-L, and CD58, and wherein the one or more viral vectors comprise a nucleic acid encoding CD86 and a nucleic acid encoding 4-1BBL and/or OX40L and transduce the myeloid cell to express CD86 and 4-1BBL and/or OX40L proteins. In some embodiments, methods of expanding tumor infiltrating lymphocytes (TILs) with aAPCs and methods of treating cancers using TILs after expansion with aAPCs are also disclosed.

Abstract: This application provides for enriched target populations oligodendrocyte precursor cells (OPCs) that can differentiate into oligodendrocytes. The target OPCs may be expanded and optionally subjected to conditions to induce their differentiation into oligodendrocytes. The target OPCs and their progeny are useful for the treatment of disease associated with demyelination of central nervous system axons.

Abstract: An object of the present invention is to provide a method for producing a mesenchymal stromal cell composition, comprising conveniently and aseptically separating high-purity amnion-derived MSCs by performing enzyme treatment only once. According to the present invention, the following are provided: a method for producing a mesenchymal stromal cell composition, comprising: performing enzyme treatment of an amnion with collagenase and thermolysin and/or dispase; and filtering the enzyme-treated amnion through a mesh; a method for producing a cryopreserved mesenchymal stromal cell composition; and a therapeutic agent comprising as an active ingredient the mesenchymal stromal cell composition for a disease selected from graft-versus-host disease, inflammatory bowel disease, systemic lupus erythematosus, liver cirrhosis, or radiation enteritis.

Abstract: This invention relates to molecules, particularly polypeptides, more particularly fusion proteins that include a serpin polypeptide or an amino acid sequence that is derived from a serpin and second polypeptide comprising of at least one the following: an Fc polypeptide or an amino acid sequence that is derived from an Fc polypeptide; a cytokine targeting polypeptide or a sequence derived from a cytokine targeting polypeptide; a WAP domain containing polypeptide or a sequence derived from a WAP containing polypeptide; and an albumin polypeptide or an amino acid sequence that is derived from a serum albumin polypeptide. This invention also relates to methods of using such molecules in a variety of therapeutic and diagnostic indications, as well as methods of producing such molecules.

Abstract: Engineered, living, three-dimensional liver tissue constructs including: one or more layers, wherein each layer contains one or more liver cell types, the one or more layers cohered to form a living, three-dimensional liver tissue construct free of pre-formed scaffold. Also disclosed are arrays and methods of making the same.

Abstract: The invention relates to polynucleotide agents targeting the complement component C5 gene, and methods of using such polynucleotide agents to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.

Abstract: A volumetric measurement system having an imaging device and a light source, where the light source is configured to illuminate the container and the sample regardless of a blockage or obstruction of the sample on at least part of the container.

Abstract: The present invention encompasses methods and compositions for the generation and use of cytotoxic T lymphocytes that target multiple viruses or that are specific for multiple tumor antigens. In specific embodiments, the generation methods employ use of certain cytokines to promote proliferation and reduce cell death in an activated T cell population and/or that employ a particular bioreactor having a gas permeable membrane.

Abstract: The invention relates to polynucleotides, particularly chimeric polynucleotides useful for optimal production of functional immunoglobulins with human idiotypes in rodents. The invention further relates to rodents comprising such polynucleotides.

Abstract: It is an object of the present invention to provide a cell culture medium capable of enhancing cell growth efficiency without using feeder cells, in particular which does not comprise serum. The present invention provides a cell culture medium which comprises growth arrest-specific 6 (GAS6) and does not comprise serum.

Abstract: A method for engineering and utilizing large DNA vectors to target, via homologous recombination, and modify, in any desirable fashion, endogenous genes and chromosomal loci in eukaryotic cells. These large DNA targeting vectors for eukaryotic cells, termed LTVECs, are derived from fragments of cloned genomic DNA larger than those typically used by other approaches intended to perform homologous targeting in eukaryotic cells. Also provided is a rapid and convenient method of detecting eukaryotic cells in which the LTVEC has correctly targeted and modified the desired endogenous gene(s) or chromosomal locus (loci) as well as the use of these cells to generate organisms bearing the genetic modification.

Abstract: This invention provides a method for stably producing airway epithelial cells from pluripotent stem cells. Specifically, the invention relates to a method for producing airway epithelial cells from pluripotent stem cells comprising steps: (1) culturing pluripotent stem cells in a medium containing activin A and a GSK3? inhibitor; (2) culturing the cells obtained in Step (1) in a medium containing a BMP inhibitor and a TGF? inhibitor; (3) culturing the cells obtained in Step (2) in a medium containing BMP4, retinoic acid, and a GSK3? inhibitor; (5) subjecting the cells obtained after Step (3) to three-dimensional culture in a medium containing a GSK3? inhibitor, FGF10, and a ROCK inhibitor; and (6) subjecting the proximal airway epithelial progenitor cells obtained in Step (5) to three-dimensional culture in a medium containing a ROCK inhibitor.

Abstract: A method for engineering and utilizing large DNA vectors to target, via homologous recombination, and modify, in any desirable fashion, endogenous genes and chromosomal loci in eukaryotic cells. These large DNA targeting vectors for eukaryotic cells, termed LTVECs, are derived from fragments of cloned genomic DNA larger than those typically used by other approaches intended to perform homologous targeting in eukaryotic cells. Also provided is a rapid and convenient method of detecting eukaryotic cells in which the LTVEC has correctly targeted and modified the desired endogenous gene(s) or chromosomal locus (loci) as well as the use of these cells to generate organisms bearing the genetic modification.

Abstract: A method for engineering and utilizing large DNA vectors to target, via homologous recombination, and modify, in any desirable fashion, endogenous genes and chromosomal loci in eukaryotic cells. These large DNA targeting vectors for eukaryotic cells, termed LTVECs, are derived from fragments of cloned genomic DNA larger than those typically used by other approaches intended to perform homologous targeting in eukaryotic cells. Also provided is a rapid and convenient method of detecting eukaryotic cells in which the LTVEC has correctly targeted and modified the desired endogenous gene(s) or chromosomal locus (loci) as well as the use of these cells to generate organisms bearing the genetic modification.

Abstract: A method for engineering and utilizing large DNA vectors to target, via homologous recombination, and modify, in any desirable fashion, endogenous genes and chromosomal loci in eukaryotic cells. These large DNA targeting vectors for eukaryotic cells, termed LTVECs, are derived from fragments of cloned genomic DNA larger than those typically used by other approaches intended to perform homologous targeting in eukaryotic cells. Also provided is a rapid and convenient method of detecting eukaryotic cells in which the LTVEC has correctly targeted and modified the desired endogenous gene(s) or chromosomal locus (loci) as well as the use of these cells to generate organisms bearing the genetic modification.

Abstract: An antisense oligomer capable of binding to a selected target on the ITGA4 gene transcript to modify pre-mRNA splicing in an ITGA4 gene transcript or part thereof.

Abstract: A method of culturing pluripotent stem cells is provided. The method includes culturing pluripotent stem cells in a pluripotent stem cell culture medium supplemented with an additive, where the additive includes a source of acetate ions, a carboxylic acid, or a physiologically acceptable salt of the carboxylic acid, or a combination of these substances, in an amount effective to maintain the pluripotent stem cells in culture in an undifferentiated pluripotent state. Also included are pluripotent stem cell culture media and methods of making such media.

Abstract: A variable diameter bioreactor vessel is provided that includes a first vessel section having a first diameter configured to hold a liquid media and biologic material, and a second vessel section having a second diameter that is greater than the first diameter such that the liquid media can be increased from a first volume to a second volume within the vessel.

Abstract: Disclosed herein are cell cultures and enriched cell populations of endocrine precursor cells, immature pancreatic hormone-expressing cells and mature pancreatic hormone-expressing cells. Also disclosed herein are methods of producing such cell cultures and cell populations.

Abstract: An antisense oligonucleotide capable of preventing or reducing exon 80 inclusion into a human COL7A1 mRNA, and methods for preventing or reducing exon 80 inclusion into a human COL7A1 mRNA.

Abstract: Provided herein is a method of diagnosing or prognosing an epilepsy or epilepsy-related disorder. Also provided herein is a method of treating an epilepsy or epilepsy-related disorder. Further provided are non-epileptic and epileptic neural stem cells and cell cultures.

Abstract: The present invention provides methods to promote the differentiation of pluripotent stem cells. In particular, the present invention provides methods to produce a population of cells, wherein greater than 10% of the cells in the population express markers characteristic of single hormonal pancreatic beta cells.

Abstract: The present invention provides methods, cell cultures and differentiation media to promote differentiation of pluripotent stem cells to pancreatic endocrine cells expressing PDX1, NKX6.1, and HB9 by culturing in a culture vessel at the air-liquid interface. The invention also provides for in vivo maturation of cells cultured at the air-liquid interface.

Abstract: The present disclosure relates to methods for producing dopaminergic cells and evaluating their functionality. When pluripotent human embryonic stem cells are cultured on plates coated with laminin-111, laminin-121, laminin-521, laminin-421, or laminin-511 in cell culture medium containing a GSK3 inhibitor and a TGF-? inhibitor as well as timely administered fibroblast growth factor, desired neural cells are produced at far higher rates. Useful cell culture kits for producing such dopaminergic cells are also described herein, as are methods of using such cells for stem cell therapy.

Abstract: Described herein are synthetic, modified RNAs for changing the phenotype of a cell, such as expressing a polypeptide or altering the developmental potential. Accordingly, provided herein are compositions, methods, and kits comprising synthetic, modified RNAs for changing the phenotype of a cell or cells. These methods, compositions, and kits comprising synthetic, modified RNAs can be used either to express a desired protein in a cell or tissue, or to change the differentiated phenotype of a cell to that of another, desired cell type.

Abstract: The object is to provide a lysis method, lysis treatment solution, detection method using an immunochromatographic device, and detection kit comprising an immunochromatographic device for detecting whether causative bacteria of mastitis are coliform bacteria or not by using milk of a livestock animal. There is provided a method for lysing coliform bacteria, which comprises the step of mixing a lysis agent containing a lytic enzyme, and at least one kind of anionic surfactant, and preferably further containing at least one kind of nonionic surfactant, with milk obtained form a livestock animal to lyse coliform bacteria existing in the milk. The lytic enzyme is preferably lysozyme.

Abstract: The present invention features a knock-in mouse comprising a mutation in an endogenous CRBN locus and methods of use thereof.

Abstract: Embodiments of the current invention include methods and compositions for regulating the activity of hormone receptors.

Abstract: Disclosed are methods of differentiating stem cells in order to obtain hepatocyte-like cells, the method comprising the steps of a) subjecting definitive endoderm to at least one epigenetic modulator to obtain hepatoblasts and b) subjecting the hepatoblasts to at least one stem cell differentiation pathway inhibitor to obtain hepatocyte-like cells; wherein steps a) and b) do not comprise the use of a growth factor. In one preferred embodiment, the epigenetic modulator may be sodium butyrate and/or DMSO and the stem cell differentiation pathway inhibitor may be SB431542 and/or DMSO. Also disclosed are hepatocyte-like cells obtained from the method and uses of these cells such as drug screening.

Abstract: Methods are provided for the production of photoreceptor cells and photoreceptor progenitor cells from pluripotent stem cells. Additionally provided are compositions of photoreceptor cells and photoreceptor cells, as well as methods for the therapeutic use thereof. Exemplary methods may produce substantially pure cultures of photoreceptor cells and/or photoreceptor cells.

Abstract: A flexible organic light emitting diode display and a manufacturing method are provided. The method includes steps of forming an active array layer and an organic light emitting display layer sequentially on a flexible substrate, forming a protective layer on the organic light emitting display layer, forming an organic layer on the protective layer, wherein a cross section of the organic layer is trapezoidal, and forming an inorganic layer on the organic layer.

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.