Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized albumin (ALB) locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized albumin locus express a human albumin protein or a chimeric albumin protein, fragments of which are from human albumin. Methods are provided for using such non-human animals comprising a humanized albumin locus to assess in vivo efficacy of human-albumin-targeting reagents such as nuclease agents designed to target human albumin.

Abstract: The present invention relates to a method for differentiating hair follicle cells into germline stem cells, germline stem cells differentiated by the method, and use of the same germline stem cells. A method of differentiating hair follicle cells into germline stem cells according to the present invention can differentiate hair follicle cells into germline stem cells using culture conditions only, without genetic modification. Capable of inducing differentiation of cells of specific individual types, such as hair follicle cells, into cells of different types such as germline cells, the present invention is therefore expected to be usefully used for the understanding of reproductive biology and the clinical application thereof.

Abstract: Compositions and methods for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL), also known as Juvenile Batten Disease, are provided herein. In certain embodiments the compositions include but are not limited to adeno-associated viral (AAV) constructs, including self-complementary adeno-associated viral (sc-AAV) constructs, that express the human gene CLN3 (or a CLN3 cDNA).

Abstract: RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.

Abstract: Provided herein is the first viable galactosyltransferase (Gal) knock-out sheep having a deletion or mutation of alpha-1,3-galactosyltransferase (GGTA1) gene and methods of making the same. Also provided are methods of screening a biological implant for stimulation of an antibody-mediated inflammatory response to a Gal antigen by implanting the biological implant into a recipient Gal knock-out animal and detecting signs of antibody-mediated inflammatory response in the recipient Gal knock-out animal. Further provided is a method of implanting a biological implant into a human subject by screening a first biological implant for signs of antibody-mediated inflammatory response in a recipient Gal knock-out animal and, upon detecting minimal or no signs of antibody mediated inflammatory response in the recipient Gal knock-out animal, implanting a second biological implant into the human subject, wherein the second biological implant is comparable to the first biological implant.

Abstract: The invention provides compositions and methods for reprogramming minimal volumes of mononuclear cells. In particular aspects, the invention provides methods and compositions for reprogramming minimal volumes of umbilical cord blood obtained from cord blood segments from cryopreserved cord blood segments.

Abstract: Provided is an agent for accelerating maturation or aging of cells. An agent for accelerating maturation or aging of cells, comprising KU-60019 as an active ingredient.

Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized PNPLA3 locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized PNPLA3 locus express a human PNPLA3 protein or a chimeric PNPLA3 protein, fragments of which are from human PNPLA3. Methods are provided for using such non-human animals comprising a humanized PNPLA3 locus to assess in vivo efficacy of human-PNPLA3-targeting reagents such as nuclease agents designed to target human PNPLA3.

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

Abstract: Provided is an exosome, which is secreted by a hybrid cell formed by antigen-presenting cells phagocytosing the cell nuclei of tumor cells. Using the strategy of macrophages phagocytosing the cell nuclei of tumor cells achieves endogenous expression of tumor antigens on macrophages, and the exosome prepared has good capabilities of targeting to lymph nodes and tumors dually.

Abstract: The present invention relates to methods for transfecting cells. In particular, the present invention relates to methods of transfecting primordial germ cells in avians, and to methods of breeding avians with modified traits.

Abstract: An isolated nucleic acid encoding a C-terminal fragment of paraspeckle component 1 (PSPC1) is disclosed. The C-terminal fragment of the PSPC1 comprises an extension of more than 10 but no greater than 131 amino acid residues with its C-terminal amino acid identical to the C-terminus of the PSPC1 sequence SEQ ID NO: 3 and exhibits a biological activity against tumor cells. The tumor cells are associated with either PSPC1 or protein tyrosine kinase 6 (PTK6), or both. The anti-tumor activity is at least one selected from the group consisting of: (a) suppressing tumor cell growth; (b) suppressing tumor cell progression; (c) suppressing tumor cell metastasis; (d) decreasing PSPC1 expression; and (e) decreasing oncogenic PTK6 expression in cytoplasm. Also disclosed is a peptide comprising a C-terminal fragment sequence of PSPC1. A reagent kit and method for predicting tumor progression, metastasis, and prognosis in a cancer patient are also disclosed.

Abstract: The present invention relates to transgenic ornamental fish, as well as methods of making such fish by m vitro fertilization techniques. Also disclosed are methods of establishing a population of such transgenic fish and methods of providing them to the ornamental fish industry for the purpose of marketing.

Abstract: The present invention relates to processes for transfecting cells. In particular, the present invention relates to processes for using CRISPR to incorporate a polynucleotide into the genome of an avian primordial germ cell (PGC).

Abstract: The invention provides isolated stem cells of non-embryonic origin that can be maintained in culture in the undifferentiated state or differentiated to form cells of multiple tissue types. Also provided are methods of isolation and culture, as well as therapeutic uses for the isolated cells.

Abstract: RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.

Abstract: Provided are methods and compositions for inducing cells of the inner ear (for example, cochlear and utricular hair cells) to reenter to cell cycle and to proliferate. More particularly, the invention relates to the use of agents that increase c-myc activity and/or Notch activity for inducing cell cycle reentry and proliferation of cochlear or utricular hair cells and/or cochlear or utricular supporting cells. The methods and compositions can be used to promote the proliferation of hair cells and/or supporting cells to treat a subject at risk of, or affected with, hearing loss or a subject at risk of, or affected with vestibular dysfunction.

Abstract: Alveolar-like macrophages and a method for generating alveolar-like macrophages from hemangioblasts is provided. The method comprises the steps of: i) culturing the hemangioblasts in a hematopoietic-inducing medium comprising vascular endothelial growth factor (VEGF), stem cell factor (SCF) and interleukin-3 (IL-3) for a sufficient period of time to generate macrophages, and ii) culturing the macrophages in an alveolar macrophage-inducing medium comprising granulocyte macrophage colony stimulating factor (GM-CSF), and optionally macrophage colony stimulating factor (M-CSF), under suitable conditions and for a sufficient period of time to yield alveolar-like macrophages.

Abstract: Non-human animals, methods and compositions for making and using the same, are provided, wherein said non-human animals comprise a humanization of a Cluster of Differentiation 274 (CD274) gene. Such non-human animals may be described, in some embodiments, as having a genetic modification to an endogenous CD274 gene so that said non-human animals express a Programmed cell death ligand 1 (PD-L) polypeptide that includes a human portion and an endogenous portion (e.g., a non-human portion).

Abstract: A genetically modified rodent is provided that comprises a modified Acvr1 gene that comprises a conditional altered exon 7 encoding R258G in antisense orientation, flanked by site-specific recombinase recognition sites, wherein the altered exon is inverted to sense orientation upon action of a recombinase, resulting in ectopic bone formation.