Abstract: According to certain embodiments of the present invention, methods for modulating the production of sialic acid in a system are provided, which comprise providing the system with a wild-type GNE-encoding nucleic acid sequence. According to such embodiments, the system may comprise a cell, muscular tissue, or other desirable targets. Similarly, the present invention encompasses methods for producing wild-type GNE in a system that comprises a mutated endogenous GNE-encoding sequence. In other words, the present invention includes providing, for example, a cell or muscular tissue that harbors a mutated (defective) GNE-encoding sequence with a functional wild-type GNE encoding sequence.

Abstract: The present invention relates to the method and use of reef coral fluorescent proteins in making transgenic red, green and yellow fluorescent zebrafish. Preferably, such fluorescent zebrafish are fertile and used to establish a population of transgenic zebrafish and to provide to the ornamental fish industry for the purpose of marketing. Thus, new varieties of ornamental fish of different fluorescence colors from a novel source are developed.

Abstract: The present invention relates to methods for producing transgenic animals using retroviral constructs engineered to carry a transgene(s) of interest.

Abstract: Targeting constructs and methods of using them are provided for differentiation-dependent modification of nucleic acid sequences in cells and in non-human animals. Targeting constructs comprising a promoter operably linked to a recombinase are provided, wherein the promoter drives transcription of the recombinase in an differentiated cell but not an undifferentiated cell. Promoters include Blimp1, Prm1, Gata6, Gata4, Igf2, Lhx2, Lhx5, and Pax3. Targeting constructs with a cassette flanked on both sides by recombinase sites can be removed using a recombinase gene operably linked to a 3?-UTR that comprises a recognition site for an miRNA that is transcribed in undifferentiated cells but not in differentiated cells. The constructs may be included in targeting vectors, and can be used to automatically modify or excise a selection cassette from an ES cell, a non-human embryo, or a non-human animal.

Abstract: RNA prepared by in vitro transcription using a polymerase chain reaction (PCR)-generated template can be introduced into a cell to modulate cell activity. This method is useful in de-differentiating somatic cells to pluripotent, multipotent, or unipotent cells; re-differentiating stem cells into differentiated cells; or reprogramming of somatic cells to modulate cell activities such as metabolism. Cells can also be transfected with inhibitory RNAs, such as small interfering RNA (siRNA) or micro RNA (miRNA), or combinations thereof to induce reprogramming of somatic cells. For example, target cells are isolated from a donor, contacted with one or more RNA's causing the cells to be de-differentiated, re-differentiated, or reprogrammed in vitro, and administered to a patient in need thereof. The resulting cells are useful for treating one or more symptoms of a variety of diseases and disorders, for organ regeneration, and for restoration of the immune system.

Abstract: The present disclosure provides methods of generating neural stem cells from differentiated somatic cells. The present disclosure also provides induced neural stem cells generated using a subject method, as well as differentiated cells generated from a subject induced neural stem cell. A subject neural stem cell, as well as differentiated cells derived from a subject neural stem cell, is useful in various applications, which are also provided in the present disclosure.

Abstract: Disclosed and claimed is a method of non-invasive immunization in an animal and/or a method of inducing a systemic immune response or systemic therapeutic response to a gene product. The skin of the animal is contacted with a non-replicative vector chosen from the group of bacterium, virus, and fungus, wherein the vector comprises and expresses a nucleic acid molecule encoding the gene product, in an amount effective to induce the response.

Abstract: The present invention relates to compositions and methods for tissue regeneration, particularly for treating skin lesions such as wounds. In one aspect, the invention provides wound healing composition characterized by the higher expression levels of phenotypic marker genes such as apolipoprotein D, matrix metalloprotease (2), collagen 3a1 and smooth muscle actin than the housekeeping gene ribosomal protein L32. The compositions and methods of the invention are useful especially for assisting the process of wound healing, particularly chronic open lesions that are slow to heal or resistant to healing.

Abstract: Synthetic surfaces suitable for culturing stem cell derived cardiomyocytes contain acrylate polymers formed from one or more acrylate monomers. The acrylate surfaces, in many cases, are suitable for culturing stem cell derived cardiomyocytes in chemically defined media.

Abstract: Methods are provided for producing a cardiomyocyte population from a mammalian pluripotent stem cell population. Aspects of the methods include using a Wnt signaling agonist and antagonist, each in minimal media, to modulate Wnt signaling. Also provided are kits for practicing the methods described herein.

Abstract: Composition based on polynucleotides extracted from natural sources for use in therapeutic treatment and/or as a therapeutic co-adjuvant in the treatment of degenerative diseases of the joints, in particular osteoarthritis.

Abstract: The present invention has an object of providing a method for producing specific cells by amplifying cells in a desired differentiation stage. The present invention provides a method for producing specific cells by inducing differentiation of cells, wherein an oncogene is forcibly expressed in cells in a desired differentiation stage to amplify the cells in the desired differentiation stage. The present invention also provides a method for producing specific cells, wherein oncogene-induced senescence (OIS) which is induced by the oncogene expressed in the cells in the desired differentiation stage is suppressed.

Abstract: Restoration or increase of inhibitory interneuron function in vivo is achieved by transplantation of MGE cells into the brain. Compositions containing MGE cells are provided as are uses to treat various diseases characterised by abnormal inhibitory interneuron function or in cases where increase inhibition may ameliorate neural circuits that are abnormally activated.

Abstract: The present invention provides methods of achieving directed evolution of viruses by in vivo screening or “panning” to identify viruses comprising scrambled AAV capsids having characteristics of interest, e.g., tropism profile and/or neutralization profile (e.g., ability to evade neutralizing antibodies). The invention also provides scrambled AAV capsids and virus particles comprising the same.

Abstract: The invention generally relates to postnatal dental stem cells and methods for their use. More specifically, the invention relates in one aspect to postnatal dental pulp stem cells, use of the cells to generate dentin, and differentiation of the cells. In another aspect, the invention relates to human postnatal deciduous dental pulp multipotent stem cells, use of the cells to generate dentin, and differentiation of the cells.

Abstract: The present disclosure describes nucleic acids, and viruses comprising such nucleic acids, for growing a toxic gene in an insect cell. These nucleic acids comprise a sequence encoding a toxic polypeptide, and an intron that interrupts the sequence, whereby the intron is spliced in mammalian cells but not in insect cells. Infection of mammalian cells but not insect cells with the nucleic acids or viruses can lead to expression of toxic levels of the toxic polypeptide in mammalian cells but not in insect cells. Viruses, such as an AAV or a baculovirus comprising a nucleic acid can be grown in insect cell lines in vitro and can be administered to a subject in need of therapy, such as a subject in need of cancer therapy.

Abstract: The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and/or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and/or single-stranded run-on mRNAs.

Abstract: A method for assessing male reproductive health is disclosed. In some embodiments, the method comprises screening a sperm sample for sperm DNA accelerated decondensation (SDAD), for sperm DNA fragmentation index (DFI) and/or for delayed sperm DNA decondensation (SDD). The method is useful in the screening and clinical management of reproductively challenged human couples and for assessing reproductive health of potential sperm donors. A method is also provided for selecting an assisted reproductive technology (ART) best suited for a particular donor sperm sample. An automated method for assessing SDAD and SDD test scores is also disclosed, as well as an automated sperm processing protocol for preparing sperm for analysis of SDAD and SDD.

Abstract: A method is disclosed for culturing pluripotent or multipotent cells in vitro, the method comprising attaching pluripotent or multipotent cells to a plurality of microcarriers to form microcarrier-cell complexes, and culturing the microcarrier-cell complexes in suspension culture in the presence of a ROCK inhibitor.

Abstract: An objective of the present invention is to provide vectors for conveniently and efficiently producing ES-like cells in which foreign genes are not integrated into the chromosome. The present inventors discovered methods for producing ES-like cells from somatic cells using chromosomally non-integrating viral vectors. Since no foreign gene is integrated into the chromosome of the produced ES-like cells, they are advantageous in tests and research, and immunological rejection and ethical problems can be avoided in disease treatments.