Abstract: The present disclosure provides compositions of matter, methods and instruments for nucleic acid-guided nickase/reverse transcriptase fusion enzyme editing of nucleic acids in live mammalian cells.

Abstract: The present invention relates to a method for inducing trans-differentiation of cardiomyocytes based on exosome, and more particularly, to a method for inducing trans-differentiation of a fibroblast into a cardiomyocyte, comprising the steps of: isolating exosomes in a culture medium during a process of differentiating a stem cell into the cardiomyocyte; culturing a fibroblast in a cardiomyocyte reprogramming medium containing the isolated exosomes; and culturing the fibroblast cultured in a cardiomyocyte differentiation medium containing the isolated exosomes.

Abstract: Compounds and compositions for the transient expression of exogenous telomerase activity in a cell are provided. The compounds and compositions, which relate to a ribonucleic acid coding for a telomerase reverse transcriptase, are useful in the extension of telomeres in cells needing such treatment. Such cells include, for example, cells that contain shortened telomeres and cells from subjects that may benefit from telomere extension, for example subjects that suffer from, or are at risk of suffering from, age-related or other illnesses. Also provided are methods of extending telomeres through the administration of the provided compounds and compositions to animal cells, either in vitro or in vivo, and kits including the compounds or compositions and instructions for use.

Abstract: Provided herein are methods for simultaneous spatio-temporal measurement of gene expression and cellular activity.

Abstract: Methods are disclosed herein for efficiently generating human induced pluripotent stem cells (iPSC) containing a nucleic acid including a doxycycline promoter operably linked to a nucleic acid encoding Cas9. These methods include transfecting a human somatic cell with a nucleic acid molecule comprising a doxycycline promoter operably linked to a nucleic acid encoding a Cas9, and constitutive promoter operably linked to a tetracycline responsive element and inducing the somatic cell to form an iPSC, thereby producing an iPSC that can undergo CRISPR/Cas9-mediated recombination at a high efficiency. The human iPSC, or a cell differentiated therefrom, is cultured in the presence of doxycycline to induce expression of the Cas9. These cells can then be used to target in any gene of interest by introducing nucleic acids encoding sgRNAs. Induced pluripotent stem cells produced by these methods are also disclosed.

Abstract: The present application provides materials and methods for treating a patient with Glycogen Storage Disease type 1a (GSD1a) both ex vivo and in vivo. In addition, the present application provides materials and methods for modulating the expression, function, and/or activity of the glucose-6-phosphatase, catalytic (G6PC) and/or the glucose-6-phosphatase (G6Pase) protein in a cell by genome editing.

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: This disclosure relates to compositions and methods of reversing senescence in T cells by interrupting vasoactive intestinal peptide (VIP) signaling and/or inhibiting phosphatidylinositol-3-kinase (PI3 kinase) inhibitor signaling and uses in managing cancer and chronic viral infections. In certain embodiments, the disclosure contemplates methods of reversing T cell senescence by mixing T cell in vitro with an agent that prevents VIP from interacting with VIP receptors and/or a PI3 Kinase inhibitor. In certain embodiments, the disclosure contemplates the expansion of senescent T cells by mixing with a PI3 kinase inhibitor, an agent that block VIP and VIP receptor signaling, a VIP degrading enzyme, and combinations thereof.

Abstract: A mold for producing a hydrogel support for a 3-dimensional (3D) cell culture, includes: an upper mold comprising a base and a plurality of upper unit molds protruding downward from the base to form accommodating portions of a female type corresponding to shapes of hydrogel supports, having through holes at bottoms thereof, and patterned at a lower portion of the base; a lower mold where a plurality of lower unit molds are patterned, the plurality of lower unit molds formed in a female type to respectively accommodate the plurality of upper unit molds protruding downward and having sealed lower portions; and an ejecting unit for separating, from the accommodating portions of the plurality of upper unit molds, the hydrogel supports that are coagulated after being inserted into the through holes respectively formed in the plurality of upper unit molds.

Abstract: The present application provides materials and methods for treating a patient with Alpha-1 antitrypsin deficiency (AATD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing the SERPINA1 gene in a cell by genome editing.

Abstract: The present disclosure relates to an improved process of organ perfusion with vitrifiable concentrations of cryoprotective agents. In one implementation, the method may include perfusing an organ with a first solution containing a vitrifiable concentration of cryoprotectant at a temperature at or above ?10° C. and perfusing the organ with a second solution containing a higher concentration of cryoprotectant than the first solution. The first solution may be adapted to vitrify at a cooling rate of less than 20° C./min, and the second solution may be adapted to vitrify at a cooling rate of less than 5° C./min. The perfusing with the second solution may begin at or above ?10° C. and cause the organ to decline in temperature to below ?10° C.

Abstract: Compositions and methods for manufacturing induced immune regulatory cells comprising induced myeloid suppressive cells including MDSCs (myeloid-derived suppressor cells), dendritic cells, macrophages, and subpopulations thereof are provided. Also provided are methods and compositions for further modifying and modulating the induced immune regulatory cells to achieve enhanced therapeutic potential in treating autoimmune disorders, hematological malignancies, solid tumors, viral infections, neurodegenerative disorders, inflammatory conditions, or GvHD.

Abstract: The disclosure relates to genetically modified bacteria, genetically modified arthropods, and methods for controlling and/or reducing arthropod populations.

Abstract: The present invention provides methods and compositions for improving efficacy of viral transduction of cells. More particularly, the present invention provides methods and materials useful for safely and reliably improving the efficiency of methods and materials useful for safely and reliably improving the efficiency of methods for transducing cells, such as human hematopoietic stem cells (HSC), with viruses and/or viral vectors. The compositions and methods are useful for therapeutic indications amenable to treatment with hematopoietic stem cell gene therapies.

Abstract: Provided are functional cell and tissue assay systems based on substrate-integrated multifunctional microelectrode arrays implementing stem cell technology. The system covers normal and pathogenic characteristics.

Abstract: The present invention relates to a method for preparing a large animal model, with peritoneal carcinomatosis, which is an abdominal cavity metastasis of tumor commonly found in advanced or recurrent solid cancer, is induced using a large animal, and the method for preparing a large animal model with peritoneal carcinomatosis comprises injecting a cancer cell line into the abdominal cavity of a piglet in which the immune function is not completed. The method for preparing a large animal model with peritoneal carcinomatosis, when a human-derived immortalized cell line is inoculated into a pig, can overcome xenograft rejection and induce peritoneal carcinomatosis, can use animals with sufficient supply and demand, and can expect the spread of peritoneal carcinomatosis through breeding.

Abstract: The invention provides a splice control module for sex-specific splicing and expression of a gene of interest. In certain embodiments, a dsx-based splice control module is used to express a lethal gene in an insect that is spliced in a sex-specific manner to impart lethality to female insects but not male insects.

Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.

Abstract: The invention discloses methods for the generation of chimaeric human—non-human antibodies and chimaeric antibody chains, antibodies and antibody chains so produced, and derivatives thereof including fully humanised antibodies; compositions comprising said antibodies, antibody chains and derivatives, as well as cells, non-human mammals and vectors, suitable for use in said methods.

Abstract: The invention relates to a cellular microcompartment comprising successively, organized around a lumen, at least one layer of pluripotent cells, an extracellular matrix layer and an outer hydrogel layer. The invention also relates to processes for preparing such cellular microcompartments.