Abstract: A method of preparing a therapeutic cell population for clinical use from a starting population of cells comprising haematopoietic stem cells, said method comprising separating a population of cells that substantially do not express CD38 but which express CD34 from the starting population of cells, and transducing the separated cell population with a vector to obtain the therapeutic cell population.

Abstract: The present invention relates to immunotherapeutic methods, and molecules and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumour-associated T-helper cell peptide epitopes, alone or in combination with other tumour-associated peptides, that serve as active pharmaceutical ingredients of vaccine compositions which stimulate anti-tumour immune responses. In particular, the present invention relates to 49 novel peptide sequences derived from HLA class II molecules of human tumour cell lines which can be used in vaccine compositions for eliciting anti-tumour immune responses.

Abstract: Methods for treating conditions by administration of placenta derived adherent stromal cells to a subject in thereof are provided. Such conditions include skeletal muscle defects, neuropathic pain, and myocardial infarction. Also provided are methods wherein the adherent stromal cells administered are cultured under 2 dimensional or 3 dimensional growth conditions. Also provided are methods in which the cells administered are at least 70% adherent cells from a maternal or fetal portion of the placenta.

Abstract: This invention relates to the forward programming of pluripotent stem cells (PSCs) into megakaryocyte (MK) progenitor cells using the transcription factors GATA1, FLI1 and TAL1. Methods of producing megakaryocyte (MK) progenitor cells and subsequently differentiating them into mature megakaryocytes are provided.

Abstract: The present invention provides compositions and methods for treating cancer in a human. The invention includes administering a genetically modified Th17 cell to express a CAR having an antigen binding domain, a transmembrane domain, and an ICOS intracellular signaling domain.

Abstract: The present invention relates in part to nucleic acids, including nucleic acids encoding proteins, therapeutics and cosmetics comprising nucleic acids, methods for delivering nucleic acids to cells, tissues, organs, and patients, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, therapeutics, and cosmetics produced using these methods, kits, and devices.

Abstract: A method of qualifying whether a cell population is a suitable therapeutic is disclosed. The method comprises: (a) incubating a population of undifferentiated mesenchymal stem cells (MSCs) in a differentiating medium comprising basic fibroblast growth factor (bFGF), platelet derived growth factor (PDGF), heregulin and cAMP for at least two days to obtain a population of differentiated MSCs; and (b) analyzing the expression of CD49 a in the differentiated MSC population, wherein an amount of CD49 a above a predetermined level indicative of the cell population being suitable as a therapeutic.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.

Abstract: The present invention relates to a method of preparing vascular endothelial cells by transforming (transdifferentiating) adult fibroblasts and a composition, which includes vascular endothelial cells prepared according to the method, for preventing and treating ischemic diseases, the method including a step of transducing adult fibroblasts with a gene. In particular, the present invention confirms that five factors, Foxo1, Er71, Klf2, Tal1, and Lmo2, induce transdifferentiation of adult fibroblasts into induced vascular endothelial cells. Furthermore, the present invention confirms that three factors, Er71, Klf2, and Tal1 induce transdifferentiation of human adult fibroblasts into induced vascular endothelial cells. The resultant induced endothelial cells enable lower limb salvaging by angiogenesis in lower limb ischemic animal models, showing that the induced endothelial cells can be effectively used for prevention or treatment of ischemic diseases.

Abstract: The present invention relates to a method for treating MI or AMI in a subject in need thereof comprising a step of administering to said subject a therapeutically effective amount of an agent capable of depleting CD8 T cells. More particularly, this present invention relates to a method for treating acute myocardial infarction by reducing the size of necrosis and limiting 10 the post ischemic left ventricular remodeling.

Abstract: The present invention provides compositions comprising an anti-CD7 chimeric activating receptor (CAR) and an anti-CD7 protein expression blocker, and methods of using such compositions in cancer therapy.

Abstract: The present invention relates to methods of enhancing proliferation and/or survival of mesenchymal precursor cells (MPC) and/or progeny derived therefrom in vitro or in vivo comprising exposing the MPC or progeny to SDF-1 or analog thereof. The invention also relates to compositions comprising isolated MPCs or progeny derived therefrom and SDF-1 or analogues thereof. The present invention also relates to using such methods and compositions for ex vivo or in vivo bone formation in mammals.

Abstract: The present invention relates to compositions and methods for diagnosing and treating diseases, disorders or conditions associated with dysregulated expression of FSHR. The invention provides novel peptides that specifically bind to Follicle-stimulation hormone receptor (FSHR).

Abstract: Novel adult liver progenitor cells (called H2Stem Cells) have been have been characterized on the basis of a series of biological activities and markers. Methods for producing H2Stem Cells allow providing such cells in the form of adherent cells and three-dimensional cell clusters in suspension that can be differentiated into cells having strong liver-specific activities and/or that can be used for treating liver diseases or for evaluating the efficacy, the metabolism, and/or toxicity of a compound.

Abstract: The invention includes compositions and methods related to multimodal therapies, e.g., for treating a cancer. A multimodal therapy described herein provides and/or administers a plurality of agents that function in a coordinated manner to provide a therapeutic benefit to a subject in need thereof, e.g., a subject having a cancer.

Abstract: The present invention provides methods for isolating human cardiac ventricular progenitor cells (HVPs), wherein cultures of day 5-7 cardiac progenitor cells are negatively selected for one or more first markers expressed on human pluripotent stem cells, such as TRA-1-60, to thereby isolate HVPs. The methods can further include positive selection for expression of a second marker selected from the group consisting of JAG1, FZD4, LIFR, FGFR3 and TNFSF9. Large populations, including clonal populations, of isolated HVPs that are first marker negative/second marker positive are also provided. Methods of in vivo use of the HVPs for cardiac repair or to improve cardiac function are also provided. Methods of using the HVPs for cardiac toxicity screening of test compounds are also provided.

Abstract: The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.

Abstract: The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell expressing a CAR having an antigen binding domain, a transmembrane domain, a CD2 signaling domain, and a CD3 zeta signaling domain. The invention also includes incorporating CD2 into the CAR to alter the cytokine production of CAR-T cells in both negative and positive directions.

Abstract: The present invention provides methods for expanding human umbilical cord blood stem cells and methods for stimulating growth factor production by cord blood stem cells using an in vitro cell culture system comprising a lithium salt. The present invention also provides in vivo methods for enhancing the survival and growth of transplanted cord blood stem cells by treating the cells with a lithium salt prior to transplantation. In vivo methods for reducing rejection of transplanted cord blood stem cells by administering a lithium salt after transplantation are also provided.

Abstract: This invention provides compositions in the form of skin substitutes comprising epithelial cells and mesenchymal cells, wherein the mesenchymal cells are not isolated from the occipital or nape region of the scalp, as well as methods for using the same.

Abstract: The present invention provides a chimeric protein having the formula: Casp-Ht1-Ht2 wherein Casp is a caspase domain; Ht1 is a first heterodimerization domain; and Ht2 is a second heterodimerization domain and wherein, in the presence of a chemical inducer of dimerization (CID), an identical pair of the chimeric proteins interact such that Ht1 from one chimeric protein heterodimerizes with Ht2 from the other chimeric protein, causing homodimerization of the two caspase domains. The invention also provides a cell comprising such a protein and its use in adoptive cell therapy.

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

Abstract: Disclosed herein are methods and compositions for determining the presence or absence of a mechanism of antimicrobial resistance in a sample.

Abstract: Nucleic acids that encode chemically-inducible fusion proteins, vectors that contain nucleic acids that encode chemically-inducible fusion proteins, chemically-inducible fusion proteins, chemically-inducible fusion protein complexes, and non-natural cells that are modified to express chemically-inducible fusion proteins to spatially and temporally control immune cell signal initiation and downstream responses for treating disease.

Abstract: Described herein are methods and devices for the long term treatment of ophthalmic disorders. Also disclosed are encapsulated cell therapy (ECT) devices that secrete a biologically active molecule and methods for using the same for the treatment of various kinds of ophthalmic disorders, including retinitis pigmentosa, geographic atrophy (dry age-related macular degeneration), glaucoma and/or macular telangiectasia.

Abstract: A T cell receptor molecule (TCR) containing an alpha chain portion and a beta chain portion wherein the alpha chain portion contains three complementarity determining regions (CDRs): CDR1?: SSYSPS CDR2?: YTSAATL CDR3?: VVSPFSGGGADGLT or comprising or consisting of SPFSGGGADGLT and the beta chain portion contains three complementarity determining regions (CDRs): CDR1?: DFQATT CDR2?: SNEGSKA CDR3?: comprising SARDGGEG or comprising or consisting of RDGGEGSETQY, or wherein up to three amino acid residues in one or more CDRs are replaced by another amino acid.

Abstract: The present invention relates to a viral vector for expression of the cell death-inducing PUMA protein, for use in gene therapy, for example for the treatment of rheumatoid arthritis. This vector consists in particular of a recombinant adenovirus expressing the gene encoding the PUMA protein and of a recombinant baculovirus containing a coxsackie-adenovirus receptor (CAR).

Abstract: A method of assisting daily living for an individual, comprising the steps of measuring a presence and/or concentration of one or more biomarkers in a sample from the individual; predicting a behavior or emotional state of the individual based on the measured presence and/or concentration of the one or more biomarkers by applying a correlation between human biological phenotype and human behavioral and/or emotional phenotype; and altering the living environment of the individual or providing information, advice or guidance to the individual based on the predicted behavior or emotional state of the individual, where the correlation between human biological phenotype and human behavioral and/or emotional phenotype comprises a correlation between the one or more biomarkers and a behavior or emotional state. A system for assisting daily living for an individual is also provided.

Abstract: The present disclosure relates generally to anti-C10orf54 antibodies, including antibody-drug conjugates comprising the antibodies, and methods of their use.

Abstract: The present disclosure relates generally to anti-C10orf54 antibodies, including antibody-drug conjugates comprising the antibodies, and methods of their use.

Abstract: Compositions and methods of promoting wound healing in a human by administering to the human mesenchymal stem cells in an effective amount.

Abstract: Provided is a method of treating coronary artery disease in a mammal, comprising administering to a region of the heart of the mammal (a) a first vector encoding one or more angiogenic proteins which induce vascularization in the heart of the mammal, and (b) a second vector encoding one or more cardio-differentiating transcription factors which induce the production of induced cardiomyocytes (iCM) in the heart of the mammal, whereby the coronary artery disease in the mammal is treated. In a preferred embodiment, the first vector is an adenoviral vector encoding VEGF and the second vector is a lentiviral vector encoding Gata4, Mef2c, and Tbx5 (GMT).

Abstract: Disclosed are immunotherapeutic compositions and the concurrent use of combinations of such compositions for the improved induction of therapeutic immune responses and/or for the prevention, amelioration and/or treatment of disease, including, but not limited to, cancer and infectious disease.

Abstract: The present invention relates to viral transformation method, particularly foamy virus-mediated transformation method. The present invention relates to the transfer of transgene into cells by the safe and efficient transfer of RNA encoding foamy components. The present invention has therefore therapeutic interest, especially in the field of gene therapy.

Abstract: The present invention describes transgenic animals with human(ized) immunoglobulin loci and transgenes encoding human(ized) Ig? and/or Ig? sequences. Of particular interest are animals with transgenic heavy and light chain immunoglobulin loci capable of producing a diversified human(ized) antibody repertoire that have their endogenous production of Ig and/or endogenous Ig? and/or Ig? sequences suppressed. Simultaneous expression of human(ized) immunoglobulin and human(ized) Ig? and/or Ig? results in normal B-cell development, affinity maturation and efficient expression of human(ized) antibodies.

Abstract: Various systems and methods are implemented for controlling stimulus of a cell. One such method is implemented for optical stimulation of a cell expressing an NpHR ion pump. The method includes the step of providing a sequence of stimuli to the cell. Each stimulus increases the probability of depolarization events occurring in the cell. Light is provided to the cell to activate the expressed NpHR ion pump, thereby decreasing the probability of depolarization events occurring in the cell.

Abstract: Disclosed herein are methods and compositions for diagnosing, treating or preventing Huntington's Disease.

Abstract: Disclosed herein are methods and compositions for providing mixtures of FGF2 isoforms that are biologically active. The biological activities include, but are not limited to, stimulation of proliferation of neural precursor cells, stimulation of proliferation of endothelial cells, stimulation of development of neural precursor cells, and stimulation of development of astrocytes.

Abstract: The present application discloses the use of light-gated cation-selective channelrhodopsins (Ch Rs) for the optogenetic control of the secretion of a polypeptide of interest in adipocytes. Engineered adipocytes comprising a channelrhodopsin (ChR) polypeptide, and/or a nucleic acid encoding same, and a secretory polypeptide precursor comprising a bioactive polypeptide and a signal peptide suitable for secretion of the bioactive polypeptide by the engineered adipocytes, and/or a nucleic acid encoding same, are disclosed. The use of such engineered adipocytes for the management or treatment of diseases/conditions in which the secretion of a polypeptide of interest is beneficial, such as the secretion of insulin in diabetic patients, is also disclosed.

Abstract: A method of producing mesenchymal stem cells from induced pluripotent stem cells in which induced pluripotent stem cells are cultured in the presence of a TGF-? inhibitor an in an atmosphere containing from about 7 vol. % to about 8 vol. % CO2 for a period of time from about 20 day to about 35 days. The cells then are transferred to a culture dish having a hydrophilic surface, and the cells are cultured in a medium containing a TGF-? inhibitor for a period of time sufficient to produce mesenchymal stem cells. Such mesenchymal stem cells are more stable and less likely to form tumors, cancers, or teratomas. Also, the induced pluripotent stem cells may be genetically engineered with at least one polynucleotide encoding a therapeutic agent and then are cultured as hereinabove described to provide genetically engineered mesenchymal stem cells that express sustained amounts of a biologically active protein or polypeptide.

Abstract: The present disclosure provides isolated monoclonal antibodies that specifically bind to LAG-3 with high affinity, particularly human monoclonal antibodies. Preferably, the antibodies bind human LAG-3. In certain embodiments, the antibodies bind both human and monkey LAG-3 but do not bind mouse LAG-3. The invention provides anti-LAG-3 antibodies that can inhibit the binding of LAG-3 to MHC Class II molecules and that can stimulate antigen-specific T cell responses. Nucleic acid molecules encoding the antibodies of the invention, expression vectors, host cells and methods for expressing the antibodies of the invention are also provided. Immunoconjugates, bispecific molecules and pharmaceutical compositions comprising the antibodies of the invention are also provided. This disclosure also provides methods for detecting LAG-3, as well as methods for treating stimulating immune responses using an anti-LAG-3 antibody of the invention.

Abstract: The present invention relates to nucleic acids encoding the novel parvoviral protein “assembly activating protein” (AAP), the encoded polypeptides, methods of producing the polypeptides, antibodies specific for AAP, the use of the nucleic acids for the preparation of the polypeptides, the use of the nucleic acids or the polypeptides for the preparation of the parvoviral particle and methods of producing parvoviral particles essentially consisting of VP3 by providing in addition to the coding sequence of the parvoviral structural protein VP3 a sequence fragment Z/a nucleic acid encoding AAP in the cell and expressing VP3 and fragment Z under control of a rep-independent promoter. Furthermore, the present invention relates to parvoviral particles essentially consisting of VP3 and/oror obtainable by the above method as well as expression cassettes comprising (i) a heterologous promoter and (ii) VP3 coding sequence and/oror fragment Z.

Abstract: The disclosed embodiments include methods to form STC and STMC for use in determining presence of cancer, and methods to detect presence of cancer. In one embodiment, A portion of a STC is stained. The STC includes normal cells and cancer cells of a type of cancer co-cultured based on at least one cell culturing factors. The at least one co-culture factors includes a type of the cancer cells being cultured, a ratio of the cancer cells to the normal cells being co-cultured, seeding density of the normal cells and the cancer cells being co-cultured, a type of cell growth supplement used to facilitate culturing the cells, and a concentration of the cell growth supplement used to facilitate co-culturing the cells. The stained portion is observed to determine presence of one or more biomarker types that indicate presence of cancer.

Abstract: The present invention relates to exosomes, loaded with genetic material and methods of producing them and to the use of such exosomes for delivering genetic material in vivo, in particular the use of such exosomes in methods of gene therapy or gene silencing.

Abstract: This disclosure relates to recombinant cellular expression of chimeric proteins with peptide sequences derived from lymphocyte receptors and uses for treating cancer. In certain embodiments, the disclosure relates to a recombinant vector comprising a nucleic acid that encodes a chimeric protein with a segment with a targeting moiety based on a variable lymphocyte receptor (VLR) capable of binding a tumor associated antigen and a segment with a T cell signal transduction subunit. In certain embodiments, the recombinant vectors are used in immune based cancer treatments.

Abstract: A method and a pharmaceutical composition for treating an atherosclerotic lesion are provided, including administering a subject in need thereof a therapeutically effective amount of a composition comprising hypoxia-cultured MSCs obtained by culturing auto- or allo-MSCs under low oxygen conditions.

Abstract: Provided herein are methods and devices for the detection of conditions or disorders by detecting altered levels of stress response pathway biomarkers. Also provided are methods and reagents for identifying panels of biomarkers associated with a condition or disorder.

Abstract: The present invention relates to Chimeric Antigen Receptors (CAR) that are recombinant chimeric proteins able to redirect immune cell specificity and reactivity toward selected membrane antigens, and more particularly in which extracellular ligand binding is a scFV derived from a BCMA monoclonal antibody, conferring specific immunity against BCMA positive cells. The engineered immune cells endowed with such CARs are particularly suited for treating lymphomas, multiple myeloma and leukemia.

Abstract: Polynucleotide constructs that express an engineered foot-and-mouth disease (FMDV) P1 precursor protein and a non-FMDV TEV protease and methods for safe and efficient recombinant production of FMDV antigens and immunogens. Recombinant production of FMDV antigens avoids the need to culture highly-infectious FMDV, while conventional culture methods for producing FMDV antigens rely on the native FMDV 3C protease which exerts toxic effects on host cells. The inventors have developed a new system that efficiently and safely processes FMDV P1 precursor without the FMDV 3C protease, thus avoiding the toxic effects associated with use of the 3C protease. The invention is also directed to the FMDV antigens and virus-like particles produced by this system as well as to FMDV vaccines, diagnostics and other biologics.

Abstract: Provided is a biological information processing method and a device, a recording medium and a program that are able to predict and control changes in the state of an organism. The expression level of molecules in an organism is measured over a specific time interval; the measured time-series data is divided into a periodic component, an environmental stimulus response component and a baseline component; constant regions of the time-series data are identified from variations in the baseline component or from the amplitude or periodic variations of the periodic component; and causal relation between the identified constant regions is identified. The relation between the external environment and variations in the internal environment is identified and from the identified causal relation between the constant regions, changes in the state of the organism are inferred.