Abstract: The invention provides an animal model for hepatocellular carcinoma and uses thereof. The genome of the animal model includes a hepatitis B virus genome and a single set of endogenous miR-122 that is partially deleted. Due to the early and high incidence of spontaneous hepatocellular carcinoma in the animal model and its abnormal fat metabolism, the animal model can be used to screen candidate agents for prevention or treatment of hepatitis B virus and hepatocellular carcinoma or other diseases or to develop uses for diagnosis and prediction of hepatocellular carcinoma.

Abstract: The invention provides compositions and methods for treating diseases associated with expression of CD33. The invention also relates to chimeric antigen receptor (CAR) specific to CD33, vectors encoding the same, and recombinant T cells comprising the CD33 CAR. The invention also includes methods of administering a genetically modified T cell expressing a CAR that comprises a CD33 binding domain.

Abstract: The present invention provides stem cells enriched with healthy functional mitochondria, pharmaceutical compositions comprising these cells and methods of use thereof for treating renal diseases, disorders and symptoms thereof where the disease may or may not be associated with acquired mitochondrial dysfunction.

Abstract: Mesenchymal stem cells (MSCs) or a pharmaceutical composition comprising a therapeutically effective amount of MSCs can be used in the treatment of atopic dermatitis (AD) in canines and felines. In a second aspect, the MSCs or a pharmaceutical composition comprising a therapeutically effective amount of MSCs can be used as an immunomodulating agent during the acute and/or the chronic phase of the AD inflammatory reaction in canines and felines diagnosed with or suffering from atopic dermatitis. In a last aspect, a pharmaceutical composition comprises peripheral blood-derived MSCs.

Abstract: The present invention relate to autologous isolated unpolarized human macrophages for use in the treatment of liver disease and macrophages for use in a method of treating fibrosis in a human in need thereof.

Abstract: Methods of gene therapy are provided for treating or preventing age-related diseases or conditions by regulating one or more functional proteins associated with age-related diseases or conditions.

Abstract: The present disclosure relates to methods of treating a patient with a cancer by administering to the patient a composition comprising CAR T cells wherein the CAR T cells comprise a CAR and the CAR comprises an E2 anti-fluorescein antibody fragment, and administering to the patient a small molecule linked to a targeting moiety by a linker. The disclosure also relates to compositions for use in such methods.

Abstract: Embodiments of the invention include systems and media for mammalian cell culture that enhance cell viability which enables biological assays and quantitative studies in tissues such as skin explants. Certain embodiments may be used in coordination with each other or may be practiced separately. An exemplary embodiment of the invention is a culture medium that includes Dulbecco's Modified Eagle's Medium, X-VIVO medium, and subcutaneous adipocyte medium. In one embodiment the culture medium includes approximately 30-60% Dulbecco's Modified Eagle's Medium by volume, approximately 30-60% X-VIVO medium by volume, and approximately 2.5-15% subcutaneous adipocyte medium by volume. Certain embodiments of the culture medium can also include recombinant human stem cell factor. Embodiments of the invention disclosed here are not limited to the brands described and encompass equivalents, as well as combinations, of the individual chemical components that make up the media described herein.

Abstract: The present disclosure provides a novel technique relating to immunological tolerance. More specifically, the present inventor found for the first time that, in a technique for inducing immunological tolerance by administering an organ transplantation patient (a recipient) a cell preparation containing cells in which anergy is induced by an inhibitor inhibiting the interaction between CD80/CD86 and CD28, the immunological tolerance continues even after the disappearance of the cells derived from the cell preparation from the recipient (infectious immunological tolerance). Further, the present inventor proved that such a cell preparation can elicit immunological tolerance against immunological rejection caused by allergy, iPS cells, etc. or cells, tissues or organs derived therefrom.

Abstract: Described herein are modified oncolytic viruses that can contain modifications in the viral genome and exogenous nucleic acids coding for proteins. The modified oncolytic virus can be utilized as a platform vector for systemic delivery. Further described herein are oncolytic viruses having an exogenous nucleic acid that codes for a protein or a functional variant thereof that enhances degradation of an extracellular matrix (ECM) associated with a tumor, wherein the protein is a membrane associated protein.

Abstract: The present invention is based, in part, on the identification of an IRE1?-XBP1-cMyc axis in NK cell immunity. The present invention provides compositions and methods for treating conditions that would benefit from modulating (e.g., upregulating or downregulating) an immune response using an agent that modulates the IRE1?-XBP1 pathway, or a composition comprising modified NK cells.

Abstract: Provided herein are recombinant antigen receptors, for example chimeric antigen receptors (CARs), that comprise modified cytoplasmic domains that provide improved signalling and thereby provide improved performance and safety. Also provided are polynucleotides encoding the recombinant antigen receptors, vectors comprising the polynucleotides, and engineered immune cells comprising the vectors and/or polynucleotides. The invention further provides methods for engineering immune cells to express the recombinant antigen receptors. Improved recombinant antigen receptor signalling is also provided by co-expressing a first recombinant antigen receptor and a second recombinant antigen receptor or co-expressing a recombinant antigen receptor and a protein involved in transducing the signal from the activated recombinant antigen receptor.

Abstract: In some aspects, the disclosure relates to compositions and methods for modulating (e.g., increasing and/or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).

Abstract: The present disclosure provides methods for re-programming effector T cells to a central memory phenotype comprising culturing the effector T cells with a histone deacetylase inhibitor (HDACi) and IL-21. Further provided are methods of treating cancer comprising administering the central memory T cells.

Abstract: A method of treating a subject having cancer is described that includes administering a therapeutically effective amount of a ?-(1,3)-(1,4) glucan to the subject. Methods of immunostimulating in a subject by administering an effective amount of a ?-(1,3)-(1,4) glucan to the subject are also described.

Abstract: Immune cell engineered to inhibit the endogenous expression of one or more of Blimp-1 and A20 and/or overexpress one or more of exogenous TCF7 and Bach2. Method of treating cancer, comprising administering the cells described herein. Method of increasing one or more of a peak fold proliferation rate, a killing efficiency, or inducing the cellular characteristics associated with naïve phenotype of an immune cell, comprising introducing an exogenous construct encoding a CAR or a TCR, and inhibiting the endogenous expression of one or more of Blimp-1 and A20, and/or introducing an exogenous construct encoding one or more of TCF7 and Bach2. Method of generating a modified immune cell, comprising introducing an exogenous construct encoding a CAR or a TCR, and inhibiting the endogenous expression of one or more of Blimp-1 and A20, and/or introducing an exogenous construct encoding one or more of TCF7 and Bach2.

Abstract: The invention includes a method of evaluating a bovine embryo by fertilizing an egg obtained from a first bovine heterozygote of a recessive lethal haplotype with sperm cells obtained from a second bovine heterozygote of the recessive lethal haplotype; producing the embryo from the fertilized egg, wherein the embryo is homozygous for the lethal haplotype; establishing a cell culture from the embryo; collecting a plurality of cultured cells; and obtaining omics data, comprising one or more features, from the plurality of cultured cells.

Abstract: The application provides gene therapies for treating monogenic forms of nephrotic syndrome.

Abstract: The present invention provides a mutated woodchuck post-transcriptional regulatory element (WPRE). In particular, the present invention relates to a mutated WPRE sequence that can efficiently express nucleotides of interest in a retroviral vector system. The present invention also relates to methods of delivering and expressing nucleotides of interest to a target cell.

Abstract: Provided is a method for preparing functional hepatic (progenitor) cells or functional small intestinal epithelial (progenitor) cells, comprising the step of culturing an isolated cell population comprising hepatic (progenitor) cells or small intestinal epithelial (progenitor) cells in the presence of an antibiotic. Also, provided is a substantially homogeneous isolated cell population comprising functional hepatic progenitor cells, wherein an expression level of CYP3A4 in the functional hepatic progenitor cells is increased by at least 5 times the expression level thereof in a HepaRG® cell line.