Abstract: Provided is a method for producing cerebral cortex neurons from pluripotent stem cells. Provided is a method for producing cerebral cortex neurons from pluripotent stem cells, comprising (i) a step of performing a suspension culture of pluripotent stem cells in a culture medium containing a TGF? inhibitor, bFGF, a Wnt inhibitor, and a BMP inhibitor, (ii) a step of performing a suspension culture of the cells obtained in the step (i) in a culture medium containing a Wnt inhibitor and a BMP inhibitor, and (iii) a step of further culturing the cells obtained in the step (ii).

Abstract: Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.

Abstract: Methods are provided, in some aspects, for differentiating pluripotent cells into midbrain dopaminergic (DA) neurons using a mono-SMAD inhibition or inhibition of SMAD signaling with only one SMAD inhibitor. In some embodiments, mono-SMAD inhibition utilizes a single inhibitor of bone morphogenic protein (BMP) for differentiating pluripotent cells into midbrain DA neurons.

Abstract: Described here are three-dimensional microenvironment niches prepared from biomaterial compositions that support growth and self renewal of stem cells. The invention also provides methods for inducing pluripotency in a somatic cell using chemical compounds, as well as methods for screening for compounds that can induce pluripotency in a somatic cell.

Abstract: The present invention relates to the prevention and/or treatment of ADA-SCID, in a patient.

Abstract: An object of the present invention is to provide a method of inducing pluripotent stem cells to differentiate into clinically applicable retinal ganglion cells. The present invention provides a method for inducing pluripotent stem cells to differentiate into retinal ganglion cells that can be used for clinical application. Such method is a method for producing retinal ganglion cells with elongated axons comprising the following steps: (a) a step of inducing pluripotent stem cells to differentiate into retinal progenitor cells via floating culture; (b) a step of inducing the retinal progenitor cells obtained in step (a) to differentiate into retinal ganglion cells via floating culture; and (c) a step of allowing axons to elongate via adhesion culture of the retinal ganglion cells obtained in step (b).

Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Abstract: The present invention relates to a method for producing an interleukin-2 protein using methylotrophic yeast. The method for producing interleukin-2 according to the present invention shows high cell growth and protein synthesis rates by use of the established optimal cell line, and produces a large amount of a protein comprising interleukin-2 by use of the established optimal culture conditions utilizing methanol that is an inexpensive carbon source. In addition, the method according to the present invention isolates and purifies the protein by a simple process. Accordingly, the method according to the present invention highly pure interleukin-2, and thus has a significant effect on the mass-production of interleukin-2.

Abstract: Provided are methods for producing compositions comprising a population of cardiomyocytes enriched for or substantially devoid of sinoatrial node-like pacemaker cardiomyocytes (SANLCM) from human pluripotent stem cells (hPSCs), and methods of use thereof.

Abstract: An in vitro human cardiac multi potent or unipotent cell that has the ability to proliferate; may be maintained in standard cardiac stem cell media; can differentiate to a progenitor, precursor, or somatic cell; has the characteristics of a cardiac stem cell, a cardiac precursor cell, or a cardiac progenitor cell; does not exhibit uncontrolled growth, teratoma formation, or tumor formation in vivo; expresses one or more markers of a multipotent, unipotent or somatic cell not characteristic of a cardiac stem cell, a cardiac precursor cell, or a cardiac progenitor cell; and is derived from the reprogramming of a somatic cell, a progenitor cell or a stem cell that exhibits at least a transient increase in intracellular levels of at least one reprogramming agent; wherein the cell comprises at least one transiently expressed polypeptide or an expression vector.

Abstract: A method of obtaining a neural multipotent, unipotent or somatic cell, including: i) providing a cell of a first type which is not a neural multipotent, unipotent or somatic cell; ii) increasing expression of at least one neural multipotent or unipotent gene regulator in the cell of a first type, to a level at which the at least one neural multipotent or unipotent gene regulator is capable of driving transformation of the cell of a first type into the neural multipotent, unipotent or somatic cell, wherein the at least one multipotent or unipotent gene regulator is Musashi1 (Msi1), Neurogenin 2 (Ngn2), or both Msi1 and Ngn2; and iii) placing or maintaining the cell in a neural cell culture medium and maintaining sufficient intracellular levels of the at least one multipotent or unipotent gene regulator for a sufficient period of time to allow a stable neural multipotent, unipotent or somatic cell to be obtained.

Abstract: The present invention relates to a pouch-like structure useful for mechanically preventing distension and/or resisting dilation of the heart and for supporting the hearts function by controllable and paracrine support of a failing heart in a mammal. The pouch-like structure is composed at least partly of engineered tissue comprising genetically engineered cells, such as genetically engineered cells other than cardiac myocytes whereby said genetically engineered cells contain a gene encoding a paracrine factor, said gene encoding the paracrine factor being under control of an inducible promoter system or a heterologous promoter system. Further, the present invention relates to a method for the preparation of the pouch-like structure for therapeutic, disease modelling, and drug development applications. In addition, the present invention relates to cells other than cardiac myocytes for use in the preparation of the pouch-like structure as described herein.

Abstract: An innovative method of collecting cord blood stem cells from an isolated mammalian non-exsanguinated or partially exsanguinated placenta by placental perfusion is described and also an easy method for safe long duration cold storage of the placenta. Placental perfusion can include perfusing the isolated placenta with a pulsatile flow of perfusion solution, for example, using a pulsatile or peristaltic pump or device. The stem cells can then be isolated from the perfusate. Significantly increased amounts of CD133+ stem cells can be collected from the perfusate. The perfusion solution can include an anticoagulant. The isolated mammalian placenta need not be treated with an anticoagulant prior to perfusing. The isolated placenta can be free from an anticoagulant prior to perfusing.

Abstract: The invention relates to methods of increasing the genetic progress of a line, breed or herd of swine through the use of sex-selected sperm cells in artificial insemination techniques. The invention also encompasses methods of artificially inseminating a swine via deep intrauterine catheter or via a laparoscopic procedure, which allow the use of reduced doses of sex-selected sperm cells.

Abstract: The invention relates to methods of increasing genetic merit of swine by establishing a plurality of mating subtypes for a line of swine, and determining a percentage of progeny that are male for each of the mating subtypes, or a percentage of progeny that are female for each of the mating subtypes, that would result, relative to a control, in an increase in genetic merit in the line; the invention further relates to sorting a sperm cell sample from a male swine in one of the mating subtypes into one or more subpopulations of sperm cells, wherein a majority of sperm cells in a subpopulation of sperm cells bear X chromosomes or Y chromosomes, and inseminating one or more female swine in the one of the mating subtypes with the subpopulation of sperm cells to achieve the percentage of progeny that are male, or the percentage of progeny that are female, determined to increase genetic merit relative to the control.

Abstract: An object of the present invention is to provide a method for producing a non-human primate model of AMD, a method for evaluating the efficacy of a test substance in the prevention or treatment of AMD using the AMD animal model produced according to this method, and a method for screening substances effective in the prevention or treatment of AMD using the aforementioned AMD animal model. The method for preparing the AMD animal model consists of administering sodium iodate into a vitreous body of a non-human primate, and the method for evaluating the efficacy of a test substance in the prevention or treatment of AMD consists of preparing a non-human primate model of AMD according to the aforementioned method for preparing an AMD animal model, and evaluating the efficacy of the test substance in the prevention or treatment of AMD using the resulting AMD animal model.

Abstract: Cancer immunotherapy using genetically engineered NK cells is enhanced by expression of recombinant co-stimulatory molecules to deliver co-stimulatory signals to a recipient host's immune cells to enhance an immune response.

Abstract: The problem of the present invention is to provide a method of efficiently producing therapeutic alternative corneal endothelial cells, particularly, a method capable of stably producing them in a condition suitable for intraocular transplantation (in the anterior chamber) in a large amount. Furthermore, the present invention aims to provide a composition for transplantation, which is preferable for intraocular administration, particularly, into the anterior chamber. A therapeutic alternative corneal endothelial cell sphere can be produced by culturing stem cells in suspension in a differentiation induction medium containing a GSK3 inhibitor, retinoic acid and a ROCK inhibitor. Addition of a viscoelastic substance during intraocular (into the anterior chamber) transplantation of the sphere or cultured corneal endothelial cells dispersed into single cells can increase the number of adherent cells after transplantation.

Abstract: The present invention relates in part to nucleic acids encoding proteins, nucleic acids containing non-canonical nucleotides, therapeutics comprising nucleic acids, methods, kits, and devices for inducing cells to express proteins, methods, kits, and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods for inducing cells to express proteins and for reprogramming and gene-editing cells using RNA are disclosed. Methods for producing cells from patient samples, cells produced using these methods, and therapeutics comprising cells produced using these methods are also disclosed.

Abstract: Described are nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These are particularly useful for applications using gene therapy.