Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after re-stimulation.

Abstract: This application discloses the compositions comprising biologically active synthetic nanoparticle constructs and methods of use thereof to modify gene expression including transcriptional activation and transcriptional repression.

Abstract: Methods and devices for lyo-processing biological materials are provided. The methods include submerging the biological molecules or cells in a buffer solution comprising trehalose, withdrawing the biological molecules or cells from the buffer solution in an environment that does not include oxygen to generate lyo-stabilized biological molecules or cells, and storing the lyo-stabilized biological molecules or cells. The devices include a motor that lowers a horizontal member having a clamp for receiving a substrate toward a solvent reservoir and raises the horizontal member having the clamp up and away from the solvent reservoir.

Abstract: Drug compositions of angiogenesis therapy contain gene coding for human prostacyclin synthase (hPGIS) synthesizing prostaglandin I2 with activities of vasodialation and/or anti-platelet aggregation; drug compositions contain adeno-associated virus (AAV) inserted with gene for angiogenesis factors. The administration of the drug compositions into the aimed treatment region results in transfer of AAV type 1-hPGIS to skeletal muscles and induces a notable expression of human PGIS gene in skeletal muscles. The PGI2 is produced by mediation of the gene expression in the muscle cells, secreted, induces vessel-protective, neovascularization and anti-platelet aggregation actions, which lead to an improvement in vascular ischemia.

Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Abstract: Methods and compositions using a CRISPR-Cas Type IIIA resulting in RNA gene knockdown and knockout in an animal.

Abstract: The present invention relates to methods of treating cancer in a human subject in need thereof. In particular, the present invention relates to treating a cancer by administering a recombinant virus which expresses one or more biotherapeutic agents in a subject, and administering to the subject a nucleotide analogue or nucleotide precursor analogue chemotherapeutic agent. The invention further relates to method for treating cancer by administering a nucleotide analogue or nucleotide precursor analogue chemotherapeutic agent and a caspase inhibitor, and, optionally, also administering a recombinant virus expressing one or more biotherapeutic agents in the subject. The invention also relates to a method for treating cancer by administering purified interferon gamma to a subject and administering to the subject a nucleotide analogue or nucleotide precursor analogue chemotherapeutic agent.

Abstract: CRISPR/CAS-related compositions and methods for treatment of Leber's Congenital Amaurosis 10 (LCA10) are disclosed.

Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after re-stimulation.

Abstract: The present disclosure provides methods and compositions related to the modification of immune effector cells to increase therapeutic efficacy. In some embodiments, immune effector cells modified to reduce expression of one or more endogenous target genes, or to reduce one or more functions of an endogenous protein to enhance effector functions of the immune cells are provided. In some embodiments, immune effector cells further modified by introduction of transgenes conferring antigen specificity, such as exogenous T cell receptors (TCRs) or chimeric antigen receptors (CARs) are provided. Methods of treating a cell proliferative disorder, such as a cancer, using the modified immune effector cells described herein are also provided.

Abstract: The invention relates to a process for obtaining a cellular sprinkling compound and to the respective method of application to provide a therapeutic treatment for skin injuries, based on the implantation, by sprinkling and/or spraying, of human mesenchymal stem cells and microvascular endothelial cells that have been pre-expanded in vitro and resuspended in a regenerative solution for cellular implantation of biocompatible biomaterials. The solution is formed by blood plasma rich in growth factors obtained from the patient to be treated and, in some cases, by medical-grade type I collagen and by medical-grade hyaluronic acid, which potentiates the regeneration, re-epithelialization, and reconstruction of skin tissue.

Abstract: A suspension useful for AAV9-mediated intrathecal and/or systemic delivery of an expression cassette containing a hIDS gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.

Abstract: Provided herein, in some embodiments, are methods, compositions and kits for controlling nucleation and assembly of molecular nanostructures, microstructures and macrostructures.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in cone cells.

Abstract: A method for dissociating cell aggregates in an agitated reactor. The method comprises providing a cell culture comprising cell aggregates in the agitated reactor, contacting the cell aggregates with a dissociation reagent, generating a dissociation force in the agitated reactor and exposing the contacted cell aggregates to the generated dissociation force under conditions sufficient to dissociate the contacted cell aggregates. The method may be used in a process for passaging cells and/or generating dissociated differentiated cells from stem and/or progenitor cells.

Abstract: The present disclosure relates generally to bacterial delivery vehicles for use in efficient transfer of a desired payload into a target bacterial cell. More specifically, the present disclosure relates to bacterial delivery vehicles with desired host ranges based on the presence of a chimeric receptor binding protein (RBP) composed of a fusion between the N-terminal region of a RBP derived from a lambda-like bacteriophage and the C-terminal region of a different RBP.

Abstract: Cells, such a T-cells, are provided that comprise cytokine receptors having increased activity in response to their ligand. For example, cell can comprise IL-2 and/or IL-15 receptors having increased surface expression or signaling activity. Cells of the embodiments have a significant growth advantage in the presence of cytokines and can be used, e.g., for enhanced adoptive cell transfer therapies.

Abstract: The present invention relates to a prefunctionalized metallic nanoparticle (10) as a standardized basic building block of biofunctionalized nanoparticles (40), having a thiol-reactive metallic nanoparticle (12) that is prefunctionalized by a bifunctional molecule (20) that consists of an anchor component (22) and a short further-functionalization stub (24). Here, it is provided that the anchor component (22) comprises one or more dithiophosphate groups, and the short further-functionalization stub (24) is adapted for the attachment of a desired biofunctionalization (30) and is selected from the group consisting of i) an unmodified standardized oligonucleotide strand (26) having 2 to 18 bases for further-functionalization with biomolecules (30) having a terminal complementary strand (36) of the standardized oligonucleotide strand (26), and ii) a 2- to 18-base-long oligonucleotide strand (50; 60) that is modified with a terminal reactive group (52; 62) for biomolecules.

Abstract: The invention relates to pharmaceutical compositions that contain COVID-19 peptide specific cytotoxic T cells, and to methods for treating or preventing COVID-19 infection.

Abstract: The present invention provides a method for assessing the risk of cancer in a subject by detecting elevated methylation level in the genomic sequence of the TET1 gene, which leads to suppressed expression of this gene. A kit and device useful for practicing such a method are also provided.