Abstract: Methods of treating hemophilia A in a subject with an F8 gene mutation, wherein the F8 gene is repaired and the resultant repaired gene, upon expression, confers improved coagulation functionality to the encoded FVIII protein of the subject compared to the non-repaired F8 gene. The invention also includes methods of inducing immune tolerance to a FVIII replacement product ((r)FVIII) in a subject having a FVIII deficiency, wherein the F8 gene mutation is repaired and the repaired gene, upon expression, provides for the induction of immune tolerance to an administered replacement FVIII protein product. The invention also includes isolated nucleic acids, vectors, recombinant viruses, cells, and pharmaceutical compositions to repair the F8 gene.

Abstract: Disclosed herein are methods for generating SC-? cells using chemically defined, completely serum free media, and isolated populations of SC-? cells for use in various applications, such as cell therapy.

Abstract: The instant disclosure relates to methods for converting mammalian definitive endoderm (DE) cells into specific tissue(s) or organ(s) through directed differentiation. In particular, the disclosure relates to formation of gastric fundus tissue and/or organoids formed from differentiated definitive endoderm.

Abstract: The present invention relates to a method of promoting skin rejuvenation in a subject. The method comprises administering to the subject an effective amount of a cellular extract of epithelial or mesenchymal stem/progenitor cells isolated from the amniotic membrane of the umbilical cord, wherein the cellular extract contains growth factors and peptides and is in the form of a supernatant into which the epithelial or mesenchymal stem/progenitor cells secrete the growth factors and peptides.

Abstract: The present invention provides methods and compositions for generating transgenic animals, including transgenic mammals, as well as plasma cells that allow for cell surface capture of secreted immunoglobulin molecules produced endogenously in the plasma cells.

Abstract: The present invention provides a mouse with liver damage, having a high degree of damage against the mouse's original hepatocytes while having a uPA gene in a heterozygous form, and a method for efficiently preparing the mouse. Specifically, the method for preparing a mouse with liver damage having the uPA gene in a heterozygous form comprises the following steps of: (i) transforming mouse ES cells with a DNA fragment containing a liver-specific promoter/enhancer and cDNA that encodes a urokinase-type plasminogen activator operably linked under the control thereof; (ii) injecting the transformed mouse ES cells obtained in step (i) into a host embryo; (iii) transplanting the host embryo obtained in step (ii) via the injection of the ES cells into the uterus of a surrogate mother mouse, so as to obtain a chimeric mouse; and (iv) crossing the chimeric mice obtained in step (iii), so as to obtain a transgenic mouse in which the DNA fragment is introduced in a heterozygous form.

Abstract: A method of selecting retinal pigmented epithelial (RPE) cells from a mixed population of cells is disclosed. The method comprises: (a) analyzing the cells of the mixed population of cells for at least one of the following parameters: (i) cells which autofluorescence above a predetermined threshold; (ii) cells which express CD81 above a predetermined threshold; and (iii) cells which scatter light perpendicular to a laser beam above a predetermined threshold; and (b) selecting cells which are positive for at least one of the parameters, thereby sorting RPE cells from a mixed population of cells.

Abstract: Provided herein are methods and compositions for editing the genome of a human T cell. In some embodiments, a heterologous T cell receptor (TCR)-? chain and a heterologous TCR-? chain are inserted into exon 1 of a TCR subunit constant gene in the genome of the T cell.

Abstract: The invention provides compositions that increase the mobilization, homing, expansion, and/or differentiation of stem cells and methods of using the same for the treatment of mammals.

Abstract: Provided herein are methods of differentiating stem cells via modulating miR-124, and the differentiated cells thereby. Also provided herein are methods for the treatment of diseases using the differentiated cells.

Abstract: Methods and compositions are provided for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring targeted excision of a sequence between two CRISPR/Cas nuclease cleavage sites or disruption of a sequence near a CRISPR/Cas nuclease cleavage site and/or measuring CRISPR/Cas-induced recombination of the CRISPR reporter with an exogenous donor nucleic acid to convert the coding sequence for a first reporter protein to the coding sequence for a different second reporter protein. Methods and compositions are also provided for making and using these non-human animals.

Abstract: Provided herein are methods for directing differentiation of human pluripotent stem cells into a three-dimensional multilayered skin composition comprising an epidermal layer, a dermal layer, and a plurality of cells capable of forming a functional hair follicle. Also provided herein are three-dimensional, multilayered engineered skin compositions and methods of using the same for drug screening, for screening compounds for effects on hair growth, and for other applications.

Abstract: Provided herein are methods and formulations for stable and prolonged storage of infective juveniles of entomopathogenic nematodes.

Abstract: The present invention relates to: a transgenic Caenorhabditis elegans in which a glutamine tRNA 5? terminus-derived fragment (Gln 5?-tsRNA) is overexpressed; a preparation method therefor; and a method for screening for aging-associated factors by using the transgenic Caenorhabditis elegans. A transgenic Caenorhabditis elegans model provided in the present invention is an animal model in which Gln 5?-tsRNA is overexpressed such that aging is inhibited. When the model of the present invention is used, anti-aging mechanisms can be easily investigated, thereby significantly contributing to various research fields such as that of developing new anti-aging drugs and screening for age-inducing materials.

Abstract: This invention relates to NK cell activation and NK cell mediated immunity, immunogenic peptides, compositions and complexes; and associated methods of treatment or prophylaxis. In particular, a peptide capable of activating NK cell-mediated immunity, the peptide comprising or consisting of the amino acid sequence XnAX2X1 wherein Xn is an amino acid sequence of between 5 and 12 residues, and X1 is any amino acid; or leucine or isoleucine; and X2 is alanine, threonine, tryptophan, or serine.

Abstract: The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) SIRP?, and methods of use thereof.

Abstract: Compositions and methods are provided for modifying a rat genomic locus of interest using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Compositions and methods for generating a genetically modified rat comprising one or more targeted genetic modifications in their germline are also provided. Compositions and methods are provided which comprise a genetically modified rat or rat cell comprising a targeted genetic modification in the rat interleukin-2 receptor gamma locus, the rat ApoE locus, the rat Rag2 locus, the rat Rag1 locus and/or the rat Rag2/Rag1 locus. The various methods and compositions provided herein allows for these modified loci to be transmitted through the germline.

Abstract: Disclosed herein are universal donor stem cells and related methods of their use and production. The universal donor stem cells disclosed herein are useful for overcoming the immune rejection in cell-based transplantation therapies. In certain embodiments, the universal donor stem cells disclosed herein do not express one or more MHC-I and MHC-II human leukocyte antigens. Similarly, in certain embodiments, the universal donor stem cells disclosed herein do not express one or more human leukocyte antigens (e.g., HLA-A, HLA-B and/or HLA-C) corresponding to MHC-I and MHC-II human leukocyte antigens, thereby rendering such cells hypoimmunogenic.

Abstract: Embodiments provided herein relate to systems for synthetically-engineered reciprocal chromosomal translocation for gene insertion into a population of organisms such as insects.

Abstract: The present invention relates to a stem cell having an immunosuppressive ability in which the expression or activity of a suppressor of cytokine signaling (SOCS) is inhibited, and a pharmaceutical composition for inhibiting immunity, which includes the stem cell. In addition, the present invention relates to a composition for inducing the immunosuppressive activity of a stem cell, including a suppressor of cytokine signaling (SOCS) expression or activity inhibitor. The inhibition of suppressor of cytokine signaling (SOCS) expression or activity, according to the present invention, may enhance the immunosuppressive ability of a stem cell, and the stem cell with enhanced immunosuppressive ability may be used as an effective cell therapeutic agent in an autoimmune disease, rejection upon organ transplantation, or an allergic disease.