Abstract: Methods for production of platelets from pluripotent stem cells, such as human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs) are provided. These methods may be performed without forming embryoid bodies or clusters of pluripotent stem cells, and may be performed without the use of stromal inducer cells. Additionally, the yield and/or purity can be greater than has been reported for prior methods of producing platelets from pluripotent stem cells. Also provided are compositions and pharmaceutical preparations comprising platelets, preferably produced from pluripotent stem cells.

Abstract: This invention presents a sequence of the virus AAV/XBP1s-HA, method and its use in the improvement of cognitive functions, of memory and of learning, as presented in the in vivo studies in FIG. 12/17 right panel.

Abstract: The present invention provides methods to promote the differentiation of pluripotent stem cells. In particular, the present invention provides methods to produce a population of cells, wherein greater than 10% of the cells in the population express markers characteristic of single hormonal pancreatic beta cells.

Abstract: The present invention provides a method for lowering blood glucose levels in an animal by transplanting a population of pancreatic endocrine precursor cells into an animal.

Abstract: Methods for production of platelets from pluripotent stem cells, such as human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs) are provided. These methods may be performed without forming embryoid bodies or clusters of pluripotent stem cells, and may be performed without the use of stromal inducer cells. Additionally, the yield and/or purity can be greater than has been reported for prior methods of producing platelets from pluripotent stem cells. Also provided are compositions and pharmaceutical preparations comprising platelets, preferably produced from pluripotent stem cells.

Abstract: The present invention relates to methods of developing genetically engineered, preferably non-alloreactive T-cells for immunotherapy. This method involves the use of RNA-guided endonucleases, in particular Cas9/CRISPR system, to specifically target a selection of key genes in T-cells. The engineered T-cells are also intended to express chimeric antigen receptors (CAR) to redirect their immune activity towards malignant or infected cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer and viral infections.

Abstract: The invention relates to induced pluripotent stem cells (iPSCs) produced from source dendritic cells (DCs). The invention also relates to synthetic DCs re-differentiated the iPSCs and which display a definitive adult phenotype rather than a primitive fetal/neonatal phenotype. The invention also relates to methods for making and methods of using the iPSCs and DCs of the invention.

Abstract: An all male Culicide mosquito population is created by knocking down its Transformer-2 gene, causing the dysfunction of X chromosome-bearing sperm, hence producing severe biased male progenies. Unlike previous methods, we recently discovered that the Tra-2 knockdown also results in female-specific zygotes lethality (XX). This art is therefore also designed to kill early female zygotes (XX) that may have survived the previous knockdown, and the all male progenies are created only when an antibiotic substance has been added into food and drink to feed mosquitoes. The strict limit of the antibiotic exposure time allows mosquito-adapted Wolbachia bacteria to survive. Selected Wolbachia bacteria may induce cytoplasmic incompatibility (CI) of up to 100%. All the progenies are therefore genetically males, which cause sterility when outcrossing with females infected by another Wolbachia strain (bidirectional CI) or are uninfected (unidirectional CI) in natural environment.

Abstract: The present disclosure provides a method for designing a set of guide RNAs for hybridizing a genomic region of interest. The present disclosure further provides methods of editing at least one genomic region of interest with at least one set of guide RNAs.

Abstract: The present invention relates to chimeric immune receptor molecules for reducing or eliminating tumors. The chimeric receptors are composed a C-type lectin-like natural killer cell receptor, or a protein associated therewith, fused to an immune signaling receptor containing an immunoreceptor tyrosine-based activation motif. Methods for using the chimeric receptors are further provided.

Abstract: Early vascular cells (EVCs), including endothelial cells and pericytes, are generated from hiPSCs. Unlike the isolated endothelial progenitor cells, the differentiated ECs mature and are functional. When encapsulated in synthetic hydrogel, EVCs respond to matrix cues and self-assembled to form three-dimensional EVCs. Moreover, these EVCs respond to hypoxic microenvironment and undergo vasculogenesis to form complex 3D networks.

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 present invention pertains to a silkworm-type biotinylated CTLD14 or sRAGE and a method for manufacturing the same. One embodiment of the present invention provides a method for manufacturing biotinylated proteins, wherein the method includes A) a step for inserting a nucleic acid molecule for coding biotin ligase and protein in a coexpressable manner into a silkworm or a living organism that imparts sugar chains that are the same as the sugar chains of the silkworm, B) a step for causing the biotin ligase and protein to be expressed by disposing the silkworm or the living organism that imparts sugar chains that are the same as the sugar chains of the silkworm to conditions with which the nucleic acid molecule will carry out expression, and C) a step for administering biotin to the living organism and obtaining the biotinylated protein.

Abstract: An object of the present invention is to solve problems in terms of stagnation of research on norovirus by providing a cultured transgenic cell or a transgenic animal in which murine norovirus (MNV) can be grown across the barrier of host specificity in mammalian cells, and providing a screening method that uses the cultured transgenic cell or the transgenic animal. The present inventors have found that MNV infection is determined in a cultured transgenic mammalian cell or a mammal possessing the cultured transgenic mammalian cell as its own cell, the cultured transgenic mammalian cell containing one or more species selected from the entirety or a portion of the murine CD300F gene and/or a CD300 family gene having an extracellular domain nucleotide sequence similar to that of the murine CD300F gene. The present inventors have solved the aforementioned problems by providing, for example, a norovirus-related drug screening method on the basis of this finding.

Abstract: Methods are provided for the simple, fast, effective and safe directed differentiation of embryonic stem cells into the mature beta cells of enriched beta clusters, wherein the beta cells rapidly and reliably secrete insulin in response to glucose levels. The cells are useful transplant therapeutics for diabetic individuals. These cells can also be used for drug screening purposes to identify factors/chemicals capable of increasing beta cell functions, proliferation, survival, and resistance to immune assault.

Abstract: Provided herein are methods and devices related to inducing a population of self-renewing or senescent stem cells, to produce one or more beneficial factors for the treatment of a disease or disorder in an individual. Also provided are compositions and methods for inducing senescence, useful for inducing senescence in a population of stem cells, in order to produce one or more beneficial factors for the treatment of a disease or disorder in an individual. Methods and devices to control and customize the production of the beneficial factors for the requirements of a disease or disorder being treated are described. Also provided are factor production units for the production of the beneficial factors, and devices for the delivery of the beneficial factors to an individual in need.

Abstract: The invention is directed to the field of gene therapy, i.e. gene delivery into target cells, tissue, organ and organism, and more particularly to gene delivery via viral vectors. The inventors showed that it is possible by chemical coupling to modulate the coupling of a ligand in the surface of the capsid of AAV, for example AAV2 and AAV3b. In particular, the present invention relates to a recombinant Adeno-Associated Virus (rAAV) vector particle having at least one primary amino group contained in the capsid proteins, chemically coupled with at least one ligand L, wherein coupling of said ligand L is implemented through a bond comprising a —CSNH— bond and an optionally substituted aromatic moiety. Particularly, the inventors tested the chemical coupling of mannose ligand on AAV2 for subretinally injection to rats.

Abstract: This disclosure provides new animal models for studying Usher syndrome and developing new therapy. The technology is implemented in pigs, and other large animals in which the ophthalmic architecture and function more closely resembles architecture and function of the human eye. The animals have a genetic modification in which all or a portion of a human gene known to cause Usher syndrome in human patients replaces the host gene. Animals can be cloned or bred to be homozygous at the targeted locus, whereupon they manifest symptoms and signs of Usher syndrome. Since a substantial portion of the targeted gene has been humanized, the animals can be used to develop and test pharmacological agents such as gene therapy that are sequence dependent.

Abstract: Disclosed herein is a composition including a recombinant nucleic acid sequence that encodes an antibody. Also disclosed herein is a method of generating a synthetic antibody in a subject by administering the composition to the subject. The disclosure also provides a method of preventing and/or treating disease in a subject using said composition and method of generation.

Abstract: The present invention relates to generation of functional beta cells from human pluripotent stem cell-derived endocrine progenitors. The present invention also relates to functional beta cells produced by said methods and uses of said beta cells.