Abstract: The present invention provides modified tamabidin 2, which is a modified biotin-binding protein comprising an amino acid sequence represented by SEQ ID NO: 2, an amino acid sequence having one or more amino acid mutations in the amino acid sequence of SEQ ID NO: 2, or an amino acid sequence having an identity of not less than 80% to the amino acid sequence of SEQ ID NO: 2 and having biotin-binding activity, wherein an asparagine residue at position 115 of SEQ ID NO: 2 is replaced with cysteine. The modified biotin-binding protein has remarkable heat resistance.

Abstract: The invention generally regards methods for providing hematopoietic cells and precursors of hematopoietic cells from a variety of cell sources, such as pluripotent stem cells or somatic cells. Also provided are therapeutic compositions including the provided hematopoietic cells and precursors of hematopoietic cells, and methods of using such for the treatment of subjects.

Abstract: Described herein are capsid proteins and adeno-associated viruses capable of targeting various types of ocular cells including bipolar and horizontal cells. Also described herein are methods of treating various ocular disorders in a subject in need thereof by administering to the subject an effective concentration of a composition comprising the recombinant adeno-associated virus (AAV) of the invention.

Abstract: Described herein are methods and composition for identifying agents that modulate nerve regeneration in vivo in extended third instar (ETI) Drosophila larvae. The methods include the use of ETI Drosophila larvae having a structural or functional disruption in one or more neurons (e.g., motor neurons) to evaluate a nerve regeneration phenotype over an extended developmental time period in the presence or absence of a test agent.

Abstract: Human progenitor cells are extracted from perivascular tissue of human umbilical cord. The progenitor cell population proliferates rapidly, and harbors osteogenic progenitor cells and MHC?/? progenitor cells, and is useful to grow and repair human tissues including bone.

Abstract: This invention provides methods to prepare and use immunostimulatory cells for enhancing an immune response. The invention provides a method for preparing mature dendritic cells (DCs), comprising the sequential steps of: (a) signaling isolated immature dendritic cells (iDCs) with a first signal comprising an interferon gamma receptor (IFN-?R) agonist and/or a tumor necrosis factor alpha receptor (TNF-?R) agonist to produce signaled dendritic cells; and (b) signaling said signaled dendritic cells with a second transient signal comprising an effective amount of a CD40 agonist to produce CCR7+ mature dendritic cells. Also provided by this invention are enriched populations of dendritic cells prepared by the methods of the invention. Such dendritic cells have enhanced immunostimulatory properties and increased IL-12 secretion and/or decreased IL-10 secretion. CD40 signaling can be initiated by one or more of polypeptide translated from an exogenous polynucleotide encoding CD40L (e.g.

Abstract: Provided herein are isolated populations of kidney cells harvested from differentiated cells of the kidney, wherein cells have been expanded in vitro. The kidney cells may include peritubular interstitial cells of the kidney, and preferably produce erythropoietin (EPO). The kidney cells may also be selected based upon EPO production. Methods of producing an isolated population of EPO producing cells are also provided, and methods of treating a kidney disease resulting in decreased EPO production in a patient in need thereof are provided, including administering the population to the patient, whereby the cells produce EPO in vivo.

Abstract: The invention describes a method for isolating one or more genetic elements encoding a gene product having a desired activity, comprising of the steps of: (a) compartmentalizing genetic elements into microcapsules; (b) expressing the genetic elements to produce their respective gene products within the microcapsules; (c) sorting the genetic elements which produce the gene product having a desired activity. The invention enables the in vitro evolution of nucleic acids by repeated mutagenesis and iterative applications of the method of the invention.

Abstract: This invention provides methods to prepare and use immunostimulatory cells for enhancing an immune response. The invention provides a method for preparing mature dendritic cells (DCs), comprising the sequential steps of: (a) signaling isolated immature dendritic cells (iDCs) with a first signal comprising an interferon gamma receptor (IFN-?R) agonist and/or a tumor necrosis factor alpha receptor (TNF-?R) agonist to produce signaled dendritic cells; and (b) signaling said signaled dendritic cells with a second transient signal comprising an effective amount of a CD40 agonist to produce CCR7+ mature dendritic cells. Also provided by this invention are enriched populations of dendritic cells prepared by the methods of the invention. Such dendritic cells have enhanced immunostimulatory properties and increased IL-12 secretion and/or decreased IL-10 secretion. CD40 signaling can be initiated by one or more of polypeptide translated from an exogenous polynucleotide encoding CD40L (e.g.

Abstract: Therapeutic and drug delivery systems are provided in the form of medical devices with coatings for capturing and immobilizing target cells such as circulating progenitor or genetically-altered mammalian cells in vivo. The genetically-altered cells are transfected with genetic material for expressing a marker gene and a therapeutic gene in a constitutively or controlled manner. The marker gene is a cell membrane antigen not found in circulating cells in the blood stream and therapeutic gene encodes a peptide for the treatment of disease, such as, vascular disease and cancer. The coating on the medical device may be a biocompatible matrix comprising at least one type of ligand, such as antibodies, antibody fragments, other peptides and small molecules, which recognize and bind the target cells. The therapeutic and/or drug delivery systems may be provided with a signal source such as activator molecules for stimulating the modified cells to express and secrete the desired marker and therapeutic gene products.

Abstract: The present invention concerns methods of treating cancer and methods of inhibiting cancer cell proliferation, particularly methods of treating breast cancer, wherein the methods comprise delivering a dominant-negative inhibitor of endogenous ErbB-2.

Abstract: A method of transplanting cells into a subject is disclosed. The method comprises transplanting the cells into the paranasal sinus of the subject or the subarachnoid cavity situated between the frontal bone of skull and the olfactory bulb of the subject. Devices for paranasal sinus transplantation and subarachnoid cavity transplantation are also disclosed.

Abstract: The present invention relates to animal model systems comprising a chimera between an avian embryo and a mammalian organism. Specifically, chimeric model systems comprising normal, diseased or genetically transformed mammalian cells and tissues transplanted into avian embryos, and uses thereof for in vivo testing of drugs and therapeutic modalities are disclosed.

Abstract: A method and virus for decreasing the levels of the disease-causing protein in Huntington's disease (mhtt) are described. Increased expression of human TXNDC10, and TXN1 in diseased cells decreases levels of toxic mhtt in cell models of disease. Genetically increasing the expression of the genes producing these proteins using viral vectors, or small molecule chemical activators of gene expression or stimulators of enzymatic activity, may be used to treat Huntington's disease in the asymptomatic carrier or affected human. Since other protein misfolding neurodegenerative diseases have many features in common with Huntington's disease, including the structure of the misfolded protein, the present method may be applicable to other protein misfolding neurodegenerative diseases.

Abstract: Recombinant cells and methods are provided that relate to the use of isolated, engineered recombinant cells to directly or indirectly treat diseases or disorders in a mammalian host such as endocrine, gastrointestinal or autoimmune disorders. A recombinant cell is provided that comprises a signal sequence and a promoter, wherein: the signal sequence is capable of regulating signal-dependent expression of a target nucleic acid in a host or is capable of regulating signal-dependent expression of a target nucleic acid in response to an environmental stimulus, the cell is derived from an enteric or a commensal bacterium, and the target nucleic acid encodes a mammalian factor that promotes normal functioning of a physiological process in the host or is effective in preventing onset, establishment, or spread of a non-infectious disease in the host. The recombinant cell is administered to the host to treat the disease or disorder.

Abstract: The invention provides genetically altered fish of the family Cyprinidae, or genus Danio, including zebrafish (Danio rerio) and host cells from these animals, where the fish have been genetically altered to lack or have a modified gene related to lipid metabolism, for example, an ApoE, ApoAI and/or LDL-R gene. In another aspect, the invention is directed to drug design or discovery using the animal or cell models of the invention and/or wild type zebrafish, and by administering an altered diet and/or environment to the animal of invention. The invention also provides methods for screening for a compound capable of ameliorating or preventing or reversing: atherosclerosis; hyperlipidemia; lipoprotein oxidation; the accumulation of lipid in a blood vessel wall; vascular inflammation associated with lipid accumulation in a blood vessel wall; acute atherosclerosis-associated events; heart attack; stroke.

Abstract: The present invention relates to methods and compositions for diagnosing a disease or disorder in a subject by introducing into cells of the subject a diagnostic gene switch construct and monitoring expression of a reporter gene. The invention further relates to methods and compositions for monitoring the progression of a disease or disorder or the effectiveness of a treatment for a disease or disorder.

Abstract: The present invention relates to the discovery of the role of Wnt1 in multiple cardiovascular processes, including cardiac repair, angiogenesis, and stimulation of endothelial progenitor cells. This discovery provides methods of using Wnt1 to treat cardiovascular disorders and injuries.

Abstract: The present invention provides lipid-based formulations for delivering, e.g., introducing, nucleic acid-lipid particles comprising an interference RNA molecule to a cell, and assays for optimizing the delivery efficiency of such lipid-based formulations.

Abstract: Disclosed herein are methods and compositions for insertion of Factor IX (FIX) sequences into the genome of a cell for treating hemophilia B.