The present invention relates to an isolated human hematopoietic stem cell or progenitor cell, transduced with a lentiviral vector which comprises a coding nucleic acid sequence encoding a functional variant of a polypeptide selected from gp9lphox, p22phox, p40phox, p47phox, p67phox and Rac2; under transcriptional control of a promoter sequence that comprises or essentially consists of the miR223 promoter sequence (SEQ ID NO 01).

The present invention features compositions and methods useful in inhibiting the expression of NFIX within a cell and thereby treating patients suffering from a hemoglobinopathy such as sickle cell disease or β-thalassemia

The invention of the present disclosure provides a method for treating a viral infection in a recipient subject suffering from or at risk of a viral infection including administering to the recipient subject a pharmaceutical composition comprising a therapeutic amount of superactivated cytokine killer T cells (SCKTCs) and a pharmaceutically acceptable carrier, and mobilizing an immune response of the recipient subject to the viral pathogen. When tested in vitro, the SCKTCs are characterized by a predominant production of T.sub.H1 dominant cytokines including IFN-γ; an IFN-γ:IL-4 ratio of at least 500:1; and at least 50% killing of target A549 cells at an effector:target ratio of 20:1. The present disclosure further provides a method of preparing a pharmaceutical composition comprising an enriched population of superactivated cytokine killer T cells (SCKTCs) wherein pulsing steps with monocyte-derived dendritic cells (DCs) loaded with alpha-GalCer achieve at least an 80% pure population of SCKTCs without positive or negative cell separation methods.

Systems and methods for associating a compound with physiological conditions are provided. A fingerprint of a compound chemical structure is obtained and inputted to a model that outputs one or more calculated activation scores. Each activation score represents a cellular constituent module in a set of modules, where each module includes a subset of cellular constituents and a first module in the set of modules is associated with the physiological condition. When the activation score for the first module satisfies a threshold criterion, the compound is identified as associated with the physiological condition. In some aspects, each activation score represents a perturbation signature associated with the physiological condition and the compound is identified when the activation score for a first perturbation signature satisfies a threshold criterion. Systems and methods for training a model that associates compounds with physiological conditions are also provided.

The present disclosure provides methods for treating hematologic malignancies in a recipient subject in need thereof comprising administering to the recipient subject an effective amount of donor myeloid progenitor cells, and an effective amount of donor umbilical cord blood (UCB) cells, wherein the UCB cells and the myeloid progenitor cells are HLA matched. In some embodiments, the donor for the myeloid progenitor cells is not related to the recipient subject and/or the donor for the UCB cells. Also disclosed herein are methods for promoting early myeloid recovery in a recipient subject following UCB transplantation.

A method for enhancing an expression of insulin like growth factor 1 receptor in a mesenchymal stem cell is provided. The method includes culturing the mesenchymal stem cell expressing insulin-like growth factor 1 receptor in a medium containing platelet-derived growth factor BB (PDGF-BB) to enhance the expression of insulin like growth factor 1 receptor in the mesenchymal stem cell.

Provided are compositions and methods for production of anti-inflammatory cytokines, growth factors, or chemokines. Provided are nucleic acids (e.g., expression vectors) that include an NFκB inflammation response element operably linked to a nucleotide sequence encoding an anti-inflammatory cytokine (e.g., IL-4). In some cases, the nucleic acid is an expression vector selected from: a linear expression vector, a circular expression vector, a plasmid, and a viral expression vector. Also provided are cells (e.g., mesenchymal stem cells—MSCs) comprising a nucleic acid that includes an NFκB inflammation response element operably linked to a nucleotide sequence encoding an anti-inflammatory cytokine. In some cases, the nucleic acid is integrated into the cell's genome. Also provided are methods for treating an individual having an inflammation-associated ailment, which can include administering an MSC to the individual, where the MSC includes an NFκB inflammation response element operably linked to a nucleotide sequence encoding an anti-inflammatory cytokine.

A bone augmentation composition and a method for making the bone augmentation. The method including hydrating an allograft comprising cortical fibers with a cell culture media, seeding the hydrated allograft with a solution of human stem cells, and culturing the stem-cell seeded allograft to grow a population of the stem cells in the seeded graft prior to freezing the bone augmentation composition for storage.

This invention is directed to, inter alia, compounds, methods, systems, and compositions for the maintenance, enhancement, and expansion of hematopoietic stem cells derived from one or more sources of CD34+ cells. Sources of CDS 4+ cells include bone marrow, cord blood, mobilized peripheral blood, and non-mobilized peripheral blood. Also provided herein are compounds of Formula I which are useful in maintaining, enhancing, and expanding of hematopoietic stem cells.

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Described herein are methods of protecting and/or promoting proliferation of intestinal epithelium by administering an Interferon (IFN) λ ligand or an IFNλ, receptor agonist to a subject in need thereof. Preferred methods utilising an IFNλ receptor agonist concern preventing or treating a gastrointestinal disease, disorder or condition, such as GVHD, inflammatory bowel disease and autoimmune and therapy-related enterocolitis.