Abstract: A gene and cell therapy using a cell fusion technology is proposed. Cells overexpressing hemagglutinin neuraminidase (HN) and fusion (F) proteins have effects of enhancing cell fusion with other cells, restoring cell damage through the cell fusion with damaged cells, and transferring a normal gene. Therefore, when a vector including genes encoding the HN and F proteins of the present invention or a cell transformed with the vector is clinically applied to neurodegenerative diseases, muscular diseases, and the like, an effect of reducing the damage of damaged cells through cell fusion can be expected.

Abstract: The present invention provides a method for producing a chimeric animal using a primed pluripotent stem cell, a tissue stem cell, a progenitor cell, a somatic cell, or a germ cell. The method for producing a chimeric animal according to the present invention comprises introducing a mammal-derived cell into the embryo of a mammal, the cell being primed pluripotent stem cell, tissue stem cell, progenitor cell, somatic cell, or germ cell.

Abstract: Forced expression of a handful of transcription factors (TFs) can induce conversions between cell identities; however, the extent to which TFs can alter cell identity has not been systematically assessed. Here, we assembled a human TFome, a comprehensive expression library of 1,578 human TF clones with full coverage of the major TF families. By systematically screening the human TFome, we identified 77 individual TFs that induce loss of human-induced-pluripotent-stem-cell (hiPSC) identity, suggesting a pervasive ability for TFs to alter cell identity. Using large-scale computational cell type classification trained on thousands of tissue expression profiles, we identified cell types generated by these TFs with high efficiency and speed, without additional selections or mechanical perturbations. TF expression in adult human tissues only correlated with some of the cell lineage generated, suggesting more complexity than observation studies can explain.

Abstract: The present invention chiefly aims to provide a process for directly inducing cardiomyocytes from somatic cells without performing artificial gene transfer, a cardiomyocyte obtained thereby, and a composition comprising a combination of chemical compounds capable of using for the said process. The present invention can include a process for producing a cardiomyocyte by inducing differentiation directly from a somatic cell, the process comprising a step of culturing the somatic cell in the presence of a MEK inhibitor and a cAMP inducer, and a cardiomyocyte obtained thereby, and then a composition for producing a cardiomyocyte by inducing differentiation directly from a somatic cell, the composition comprising a MEK inhibitor and a cAMP inducer. The cardiomyocytes obtained according to the present invention are useful in regenerative medicine and the like.

Abstract: The present application provides genetically modified non-human animals and methods for producing heavy chain-only antibodies (HcAbs), wherein the genetically modified non-human animal comprises a germline genome comprising an engineered immunoglobulin heavy chain (IgH) allele at an endogenous IgH locus, wherein the engineered IgH allele lacks functional gene segments encoding CH1 domains of all endogenous IgG subclasses. In some embodiments, a genetically modified mouse is provided, comprising an engineered IgH allele that lacks a functional endogenous gene segment encoding C?3, C?1, C?2b and CH1 exon of C?2c. Further provided are HcAbs or derivatives thereof produced by the genetically modified non-human animals.

Abstract: The invention provides for AAV vectors expressing the ANO5 gene and antioxidant therapy as methods of inducing muscle regeneration and a method of treating muscular dystrophy.

Abstract: Method for expanding stemness and differentiation potential of pluripotent cells. The invention is based on the finding that increasing micro RNA-203 levels in induced pluripotent stem (iPSCs) or embryonic stem (ESCs) cells improves the quality cell fate potential and ability of these cells to differentiate into multiple cell lineages and to reach further maturation properties without interfering with their self-renewal properties. This effect is mediated through the mi R-203-dependent control of de novo DNA methyltransferases Dnmt3a and Dnmt3b, which in turn regulate the methylation landscape of pluripotent cells. The effect can be achieved by overexpression of micro RNA-203 or by adding micro RNA-203 or analogues thereof to the cell culture medium and can be observed using a variety of cellular and in vivo models.

Abstract: The present invention provides a non-naturally occurring dendritic-like myeloid leukaemia cell according to ATCC Patent Deposit Designation PTA-123875, and methods and kits utilising such cells.

Abstract: Described is the efficient and robust generation of oligodendrocyte progenitor cells (OPCs) and oligodendrocytes from pluripotent stem cells (PSCs). The protocols provided recapitulate the major steps of oligodendrocyte differentiation, from neural stem cells to OLIG2+ progenitors, and then to O4+ OPCs, in a significantly shorter time than the 120-150 days required by previous protocols. Furthermore, O4+ OPCs are able to differentiate into MBP+ mature oligodendrocytes in vitro, and to myelinate axons in vivo when injected into immuno-compromised Shiverer mice, providing proof of concept that transplantation of PSC-derived cells for remyelination is technically feasible.

Abstract: Provided is an epithelial tissue stem cell derived from an adult, which lacks at least one tumor suppressor gene.

Abstract: A method for providing a sub-population of stem cell or plurality of stem cells by determining or modulating GSTT1 expression level or genotype is disclosed together with uses of the stem cells.

Abstract: An immunoresponsive cell, such as a T-cell expressing (i) a second generation chimeric antigen receptor comprising: (a) a signalling region; (b) a co-stimulatory signalling region; (c) a transmembrane domain; and (d) a binding element that specifically interacts with a first epitope on a target antigen; and (ii) a chimeric costimulatory receptor comprising (e) a co-stimulatory signalling region which is different to that of (b); (f) a transmembrane domain; and g) a binding element that specifically interacts with a second epitope on a target antigen. This arrangement is referred to as parallel chimeric activating receptors (pCAR). Cells of this type are useful in therapy, and kits and methods for using them as well as methods for preparing them are described and claimed.

Abstract: Nucleases and methods of using these nucleases for expressing an antibody from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.

Abstract: The technology described herein relates to compositions and methods for the generation of a primordial NK/T cell, where the produced NK/T primordial cell can subsequently differentiate into T cells or NK cells at an extremely high efficiency and reproducibility. Other aspects relate to genetically modified iPSC cell lines, and method of their use to generate iPSC-derived NK/T primordial cells with a very high yield and high efficiency.

Abstract: The object of the present invention is to provide a method for incorporating an arbitrary protein, lipid, carbohydrate, or nucleic acid into an exosome. The object can be solved by a method for preparing an exosome, comprising the steps of: (a) adding a biological toxin having a perforating activity to a medium containing cells and incubating the mixture, (b) adding ATP and incubating the mixture, and (c) adding a medium containing calcium ion and incubating the mixture.

Abstract: The invention relates to culturing motor neuron cells together with skeletal muscle cells in a fluidic device under conditions whereby the interaction of these cells mimic the structure and function of the neuromuscular junction (NMJ) providing a NMJ-on-chip. Good viability, formation of myo-fibers and function of skeletal muscle cells on fluidic chips allow for measurements of muscle cell contractions. Embodiments of motor neurons co-cultures with contractile myo-fibers are contemplated for use with modeling diseases affecting NMJ's, e.g. Amyotrophic lateral sclerosis (ALS).

Abstract: Provided is an aquacultured crustacean having improved umami, uses thereof, and a method for producing the same. The crustacean does not burrow in sand, contain glycine and alanine, and has 2400 mg or more of free amino acids per 100 g of abdominal muscle. The content of glycine is 550 mg or more per 100 g of abdominal muscle, and the content of alanine is 140 mg or more per 100 g of abdominal muscle.

Abstract: The present invention provides robust, streamlined, reproducible and highly efficient eukaryotic cell transfection systems and related methods. The highly-efficient systems and methods of the present invention reduce the number of steps required to transfect cells and reduce, e.g., eliminate, the need for specialized equipment. In particular, the systems and related methods afford the ability for streamlining transfection while retaining and improving robust and reproducible transfection efficiencies, cell viability, and/or protein production. Furthermore, the highly-efficient systems and methods of the present invention for transfecting eukaryotic cells also eliminate the need for any specialized or complicated preparation of exogenous nucleic acid, which makes available high throughput and/or large scale transfection.

Abstract: Disclosed are non-naturally occurring zebrafish, such as transgenic zebrafish, which comprise a mutation in the rhodopsin (rho) gene. Also disclosed are methods of identifying compounds useful in treating retinal-specific defects and disorders, such as degeneration. Further disclosed are methods of identifying mutations in the rhodopsin gene that can cause retinal-specific defects.

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.