Abstract: A method of transforming human cells into mechanosensory hair cells (MHCs), such as inner hear hair cells in the cochlea and vestibular organs, can include: causing human Wharton's jelly cells (hWJCs) to increase expression of or biological function of HATH1 so as to transform the hWJCs into MHCs. The method can include; administering a nucleic acid that encodes HATH1 to the hWJCs; causing inhibited expression of or biological function of HES1 and/or HES5 in the hWJCs; administering a nucleic acid that inhibits HES1 and/or a nucleic acid that inhibits HES5 to the hWJCs; causing inhibited expression of or biological function of HES1 and/or HES5 in the WJCs by administering a nucleic acid that inhibits HES1 and/or a nucleic acid that inhibits HES5; nucleic acids are administered includes a sequence of SEQ ID NO: 2, SEQ ID NO: 3, and/or SEQ ID NO: 4.

Abstract: The present invention provides methods to promote the differentiation of pluripotent stem cells into insulin producing cells. In particular, the present invention provides a method to produce a population of cells, wherein greater than 80% of the cells in the population express markers characteristic of the definitive endoderm lineage.

Abstract: Transgenic pigs that express one or more non-porcine cytosine deaminases are described as well as methods of making and using such pigs.

Abstract: Disclosed herein are cell cultures comprising dorsal and/or ventral PDX1-positive foregut endoderm cells and methods of producing the same. Also disclosed herein are cell populations comprising substantially purified dorsal and/or ventral PDX1-positive foregut endoderm cells as well as methods for enriching, isolating and purifying dorsal and/or ventral PDX1-positive foregut endoderm cells from other cell types. Methods of identifying differentiation factors capable of promoting the differentiation of dorsal and/or ventral PDX1-positive foregut endoderm cells, are also disclosed.

Abstract: Chimeric proteins are expressed, secreted or released by a bacterium to immunize against or treat a parasite, infectious disease or malignancy. The delivery vector may also be attenuated, non-pathogenic, low pathogenic, or a probiotic bacterium. The chimeric proteins include chimeras of, e.g., phage coat and/or colicin proteins, bacterial toxins and/or enzymes, autotransporter peptides, lytic peptides, multimerization domains, and/or membrane transducing (ferry) peptides. The active portion of the immunogenic chimeric proteins can include antigens against a wide range of parasites and infectious agents, cancers, Alzheimer's and Huntington's diseases, and have enhanced activity when secreted or released by the bacteria, and/or have direct anti-parasite or infectious agent activity. The activity of the secreted proteins is further increased by co-expression of a protease inhibitor that prevents degradation of the effector peptides.

Abstract: The present invention relates to compositions and methods for maintaining undifferentiated pluripotent stem cell cultures.

Abstract: The present invention relates to treating and preventing pain. More particularly the present invention demonstrates the involvement of K2P potassium channels in the antalgic effect of morphine. The present invention therefore provides a screening method for identifying antalgics.

Abstract: The invention relates to microcapsule comprising at least one fat-soluble active substance selected from provitamins, vitamins and esters thereof, monounsaturated fatty acids, polyunsaturated fatty acids (PUFA's), carotenoids and benzoquinones embedded in a matrix comprising a hydrocolloid and optionally one or more other matrix components, wherein the content of active substance(s) is from 30 to 60% of total weight of the microcapsule, and wherein the ratio between said fat-soluble active substance(s) and said hydrocolloid is at least 4:1, as well as a process for preparing such microcapsules. The microcapsules of the invention may be used for the preparation of tablets, food products and other products including an active substance.

Abstract: The present invention relates to a transgenic animal model system based on the development of transgenic mice bearing components of the human immune system. Specifically, the invention relates to a Flk2 deficient Rag?/??c?/? transgenic mouse and the engraftment of said mouse with human hematopoietic stem cells. The present invention further provides methods for increasing the numbers of functionally competent human dendritic cells and the hematopoietic targets cells that they interact with in said transgenic mouse through the administration of Flk2L. The transgenic animal model system of the invention may be used for testing human vaccine candidates, for screening potential immune adjuvants and for developing novel therapeutics.

Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.

Abstract: Provided are methods for activating an antigen-presenting cell and eliciting an immune response by inducing an inducible pattern recognition receptor adapter, or adapter fragment, and CD40 activity. Also provided are nucleic acid compositions comprising sequences coding for chimeric proteins that include an inducible CD40 peptide and an inducible pattern recognition receptor adapter or adapter fragment.

Abstract: According to the invention there is provided methods for inducing pluripotent stem cells in vitro, comprising introducing a gene or polypeptide of a nuclear receptor and one or more gene or polypeptide selected from the group consisting of Sox, Krüppel-like factor or the myc family, to cells in vitro. The present invention also provides vectors and compositions for producing the same and methods for using the induced pluripotent stem cell for treating a patient in need of a pluripotent stem cell treatment.

Abstract: A process for preparing information that identifies a compound as capable of perturbing the epithelium in a D. melanogaster comprising the steps of: i) obtaining a D. melanogaster which is genetically unmodified except that the D. melanogaster optionally comprises at least one nucleotide sequence encoding a reporter polypeptide operably linked to a promoter of an endogenous protein; ii) contacting the D. melanogaster with the compound; and iii) determining whether there is a difference between the epithelium of the D. melanogaster of ii) and the epithelium of a corresponding D. melanogaster not contacted with the compound, wherein the presence of a difference between the epithelium of the D. melanogaster contacted with the compound and the epithelium of a corresponding D. melanogaster not contacted with the compound identifies the compound as a compound that is capable of perturbing the epithelium in a D. melanogaster.

Abstract: This disclosure provides for compositions and methods for the use of nucleic acid-targeting nucleic acids and complexes thereof.

Abstract: The present invention provides muscle-derived progenitor cells that show long-term survival following transplantation into body tissues and which can augment soft tissue following introduction (e.g. via injection, transplantation, or implantation) into a site of soft tissue. Also provided are methods of isolating muscle-derived progenitor cells, and methods of genetically modifying the cells for gene transfer therapy. The invention further provides methods of using compositions comprising muscle-derived progenitor cells for the augmentation and bulking of mammalian, including human, soft tissues in the treatment of various cosmetic or functional conditions, including malformation, injury, weakness, disease, or dysfunction. In particular, the present invention provides treatments and amelioration for dermatological conditions, gastroesophageal reflux, vesico-ureteral reflux, urinary incontinence, fecal incontinence, heart failure, and myocardial infarction.

Abstract: Factor Xa variants and methods of use thereof are disclosed.

Abstract: The invention provides compositions and methods for delivering agents to localized regions, tissues, or organs in vivo by conjugating agent-loaded nanoparticles to cells having homing capability. The agents may be therapeutic or diagnostic agents such as cancer chemotherapeutic agents and imaging agents respectively.

Abstract: The present invention relates to polypeptides having phytase activity. These polypeptides have an amino acid sequence which has at least 70% identity to either of three phytases derived from the bacterium Buttiauxella, and which comprises at least one of the following amino acids at the position indicated: 119N, 120L, and/or 121E. These phytases have an improved specific activity. Additional specific amino acid substitutions are also disclosed which characterize and distinguish additional phytases of the invention having improved properties such as temperature and/or pH stability, pH activity profile, temperature activity profile, substrate profile, improved performance in animal feed in vitro or in vivo. The invention also relates to isolated polynucleotides encoding the polypeptides, nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods for producing and using the polypeptides.

Abstract: The present invention provides for TLR agonist conjugates (compounds) and compositions, as well as methods of using them. The compounds of the invention are broad-spectrum, long-lasting, and non-toxic combination of synthetic immunostimulatory agents, which are useful for activating the immune system of a mammal, preferably a human and can help direct the pharmacophore to the receptor within the endosomes of target cells and enhance the signal transduction induced by the pharmacophore.

Abstract: A transgenic silkworm system for recombinant glycoprotein production is provided.