Abstract: The present invention relates to activated lymphocytes comprising cytokine-induced killer cells in which CD8+CD56+NKG2D+ cells are present at a proportion of 20% or more, and a preparation method therefor, and more particularly, to activated lymphocytes comprising cytokine-induced killing cells which have high tumor cell killing abilities and growth rates and are almost free of side effects because they do not require the combined administration of interleukin-2, and a preparation method therefor.

Abstract: RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 variants are provided. Delivery vehicles such as recombinant adeno-associated viruses deliver DNAs encoding RNAs that inhibit the expression of the dynamin-1 variants. The methods treat, for example, developmental and epileptic encephalopathies.

Abstract: There is provided method for making a cell composition which comprises step of transducing a population of cells with a mixture of at least two viral vectors, wherein at least one vector comprises a nucleic acid sequence which encodes a chimeric antigen receptor (CAR); and wherein at least one vector comprises a nucleic acid encoding an activity modulator which modulates the activity of the CAR, of a cell expressing the CAR, or of a target cell. There is also provided a cell composition made by such a method and its use in the treatment of diseases such as cancer.

Abstract: The disclosure provides methods of treating a malignancy comprising administering an effective dose of a chimeric antigen receptor genetically modified T cell immunotherapy and methods for manufacturing such immunotherapy. Some aspects of the disclosure relate to methods of determining objective response of a patient to a T cell immunotherapy based on the levels of attributes prior to and after administration of the immunotherapy to the patient.

Abstract: The present disclosure relates to engineered T cells and methods of making and using the same, as well as reagents for making the engineered T cells.

Abstract: Described are alcohol-induced liver cancer model mice, methods of generating the alcohol-induced liver cancer model mice, and methods of using the alcohol-induced liver cancer model mice. Methods of treating alcoholic liver disease, including hepatocellular carcinoma, are also described.

Abstract: Provided include compositions, methods, and systems for modulating the expression, function, and/or activity of a target gene, for example a blood-clotting protein such as Factor VIII (FVIII), in a cell by genome editing. Also provided include compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition, e.g., Hemophilia A, employing ex vivo and/or in vivo genome editing.

Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized coagulation factor XII (F12) locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized F12 locus express a human coagulation factor XII protein or a chimeric coagulation factor XII protein, fragments of which are from human coagulation factor XII. Methods are provided for using such non-human animals comprising a humanized F12 locus to assess in vivo efficacy of human-coagulation-factor-XII-targeting reagents such as nuclease agents designed to target human F12. A short isoform of F12 that is produced locally in the brain, and methods of using the short isoform, are also provide.

Abstract: A composition for regeneration of skeletal muscle includes a nerve cell secretome or an isolate thereof. An implantable therapeutic material for regeneration of skeletal muscle includes a hydrogel and nerve cells encapsulated within the hydrogel, wherein at least some of the nerve cells are living. A method of regenerating skeletal muscle includes applying the composition or implanting the implantable material adjacent to skeletal muscle myoblasts and/or myogenic cells to regenerate skeletal muscle therefrom.

Abstract: Described herein are immunoresponsive cells which are useful for their preventive and therapeutic potential against autoimmune diseases and rejections of solid organ transplants.

Abstract: The present disclosure provides methods for assessing and optimizing cellular quality of a cell-based therapy that is being produced in an automated cell engineering system. The methods suitably include monitoring molecular characteristics of the cells before, during, and after the automated process to provide feedback to the process parameters. In embodiments, the cells being produced are Chimeric Antigen Receptor (CAR) T-cells.

Abstract: The relates, in some aspects, to antisense oligonucleotide compositions and methods for modifying pre-mRNA splicing in a DYSF gene using the same. In some embodiments, the DYSF gene comprises a novel mutation that results in a pseudoexon between exons 50 and 51.

Abstract: Disclosed herein is a method of providing an anti-cancer immunity in a subject with a bone metastatic cancer. The method involves co-administering to the subject an effective amount of a gamma-delta T cell stimulating agent and an effective amount of a ?? CAR T cell that binds a tumor antigen, such as a prostate antigen for bone metastatic prostate cancer, or a breast cancer antigen for bone metastatic breast cancer. Also disclosed herein is a recombinant T cell that expresses a gamma-delta T cell receptor (TCR) and a chimeric antigen receptor (CAR) polypeptide.

Abstract: Described herein are immunoresponsive cells which are useful for their preventive and therapeutic potential against autoimmune diseases and rejections of solid organ transplants.

Abstract: The technology described herein relates to compositions and methods of generating endothelial niche cells. Embodiments of the technology described herein comprise compositions, kits, vectors, and methods related to generating or engineering endothelial niche cells. One aspect comprises a method to generate/engineer endothelial niche cells, comprising expressing one or more transcription factors in an endothelial cell, wherein the one or more transcription factors are from the Ets family, the Sox family, and/or the Nuclear Hormone (NHR) family.

Abstract: The invention refers to a method for culturing cells in a substrate in which a chamber having at least one side wall, a bottom, and a top is formed, comprising introduction of cells into the chamber, tilting of the substrate such that the cells accumulate on a side wall of the chamber, and holding the substrate in the tilted orientation such that the cells form a three-dimensional cell aggregate.

Abstract: Described herein are compositions and methods for improving homology directed repair (HDR) efficiency and reducing homology-independent integration following introduction of double strand breaks with engineered nucleases. Additionally, modifications to double stranded DNA donors to improve the donor potency and efficiency of homology directed repair following introduction of double stranded breaks with programmable nucleases.

Abstract: The present disclosure relates generally to methods of producing rejuvenated T cells, comprising, contacting T cells with at least one reprogramming factor and reactivating the contacted cells; and compositions and methods of using same. The present disclosure also describes cell populations prepared according to methods described herein. The disclosure also provides for methods of treating patients using cell populations prepared by the methods described herein.

Abstract: A method of generating multipotent stem cells from adult human peripheral blood cells by isolating the peripheral blood insulin-producing cells and exposing them to adult peripheral blood-derived mitochondria. Adult peripheral blood insulin-producing cells (PB-IPC) are isolated from adult peripheral blood by adherence to a hydrophobic surface with a positive charge, such as a Petri dish. Once the PB-IPC are isolated, mitochondria derived from adult peripheral blood are applied to the isolated PB-IPC. The mitochondria are then taken in by the PB-IPC and enter the nuclei of the PB-IPC, allowing the cells to be reprogrammed, transforming PB-IPC into multipotent stem cells and giving rise to three germ layer-derived cells. Additionally, PB-IPC give rise to functional CD34+ hematopoietic stem cell (HSC)-like cells after treatment with adult peripheral blood-derived mitochondria.

Abstract: The present invention relates to a method for enhancing the ability of regulatory T cells (Tregs) to suppress immune responses comprising increasing FOXP3 expression in a population of Tregs.