Abstract: Provided are antibodies, and fragments, derivatives, and nanoparticle conjugates thereof, particularly humanized derivatives thereof, which bind to tumor antigens. Also provided are nucleic acid molecules encoding chimeric antigen receptors (CARs) that bind to tumor antigens, polypeptides and CARs encoded by the nucleic acid molecules, vectors and host cells that include the nucleic acid molecules, methods of making the same, and methods for using the same to generate a persisting population of genetically engineered T cells in a subject, expanding a population of genetically engineered T cells in a subject, modulating the amount of cytokine secreted by a T cell, reducing the amount of activation-induced calcium influx into a T cell, providing an anti-tumor immunity to a subject, treating a mammal having a MUC1-associated disease or disorder, stimulating a T cell-mediated immune response to a target cell population or tissue in a subject, and imaging a MUC1-associated tumor.

Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.

Abstract: In one aspect, separation media are described herein operable for removing one or more water contaminants including NOM and derivatives thereof. Briefly, a separation medium includes a nanoparticle support and an oligomeric stationary phase forming a film on individual nanoparticles of the support, the film having thickness of 1 to 100 nm. In some embodiments, oligomeric chains of the stationary phase are covalently bonded to the individual nanoparticles.

Abstract: This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.

Abstract: The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to optimized polynucleotide sequences encoding Factor IX proteins, vector comprising the same, and methods of using these compositions to treat a bleeding disorder.

Abstract: Described herein are methods for improving or maintaining the barrier integrity of gingival tissue comprising administering an oral care composition comprising a curcurninoid, to the oral cavity of a subject in need thereof.

Abstract: Active surface devices for and methods of providing dried reagents in microfluidic applications is disclosed. In one example, the active surface devices include one or more dried reagent spots in relation to an active surface in the reaction (or assay) chamber thereof. In another example, the active surface devices include a dried reagent coating on the surfaces of the reaction (or assay) chamber including the active surface. In one example, the presently disclosed active surface devices are micropost-based active surface devices for providing active mixing therein. Further, a method of forming a dried reagent spot in the active surface devices is provided. Further, a method of forming a dried reagent coating in the active surface devices is provided. Further, a method of using the active surface devices for providing dried reagents in microfluidic applications is provided.

Abstract: The presently disclosed subject matter relates generally to a highly ionically conductive solid electrolyte membrane and to batteries comprising such solid electrolyte membrane.

Abstract: The description provides a molecular switch comprising at least two nanoparticles, wherein a first nanoparticle comprises DNA and/or RNA oligonucleotides, and a second nanoparticle which is complementary to the first nanoparticle comprises reverse complementary DNA and/or RNA oligonucleotides of the first nanoparticle; wherein the complementary nanoparticles interact under physiological conditions leading to thermodynamically driven conformational changes in the first and second nanoparticles leading to their re-association to release one or more duplexes comprising said DNA and/or RNA oligonucleotides and the reverse complementary DNA and/or RNA oligonucleotides, and wherein the nanoparticles are not rings and have no single stranded toeholds.

Abstract: This invention relates to polynucleotides comprising a nucleotide sequence encoding a PPT1 polypeptide or a fragment thereof, vectors (viral or non-viral vectors) comprising the same, and methods of using the same for delivery of the open reading frame to a cell or a subject and to treat infantile neuronal lipofuscinosis (infantile Batten disease). The polynucleotides comprise an optimized CLN1 open reading frame.

Abstract: This invention relates to SARS-CoV-2 viruses adapted with nanoluciferase reporter molecules and mouse-adapted SARS-CoV-2 viruses, compositions including the same and methods of use thereof.

Abstract: The invention relates to chimeric AAV capsids targeted to the central nervous system, virus vectors comprising the same, and methods of using the vectors to target the central nervous system. The invention further relates to chimeric AAV capsids targeted to oligodendrocytes, virus vectors comprising the same, and methods of using the vectors to target oligodendrocytes.

Abstract: This invention relates to polynucleotides comprising optimized aspartylglucosaminidase (AGA) open reading frames and vectors and cells comprising the same. The invention further relates to methods of using the same for delivery of the open reading frame to a cell or a subject and methods for treating aspartylglucosaminuria (AGU) in a subject.

Abstract: The invention relates to compounds of Formula (I), salts thereof, combinations, pharmaceutical compositions thereof, as well as therapeutic methods of treatment and prevention.

Abstract: Compositions and methods of transplanting cells by grafting strategies into solid organs (especially internal organs) are provided. These methods and compositions can be used to repair diseased organs or to establish models of disease states in experimental hosts. The method involves attachment onto the surface of a tissue or organ, a patch graft, a “bandaid-like” covering, containing epithelial cells with supporting early lineage stage mesenchymal cells. The cells are incorporated into soft gel-forming biomaterials prepared under serum-free, defined conditions comprised of nutrients, lipids, vitamins, and regulatory signals that collectively support stemness of the donor cells. The graft is covered with a biodegradable, biocompatible, bioresorbable backing used to affix the graft to the target site. The cells in the graft migrate into and throughout the tissue such that within a couple of weeks they are uniformly dispersed within the recipient (host) tissue.

Abstract: Methods and systems for using encapsulated microbubbles to process biological samples are disclosed. According to one aspect, a method for using encapsulated microbubbles to process a biological sample includes creating a mixture comprising encapsulated microbubbles mixed with a biological sample and adding activation energy to the mixture to cause at least some of the microbubbles to oscillate or burst and thereby process the sample, including effecting cell lysis, shearing DNA, and/or performing tissue dispersion.

Abstract: Described herein, inter alia, are compositions and methods for modulating mu opioid receptor activity.

Abstract: The present invention relates to compositions and methods for the delivery of agents to a subject, particularly to the central nervous system (CNS).

Abstract: A process for chemically recycling polyethylene terephthalate (PET) which utilizes a microwave absorber to optimize glycolytic depolymerization of PET via microwave irradiation. The method of chemically degrading PET to its reactive intermediate, bis(2-hydroxyethyl) terephthalate (BHET), is carried out by: (a) combining PET with ethylene glycol and a catalytic system comprising a catalyst and a microwave absorber to produce a heterogeneous reaction mixture; and then (b) heating by microwave irradiating the reaction mixture to a temperature sufficient to produce a reaction product comprising BHET. The BHET monomer then can be purified and re-polymerized to form new, virgin PET.

Abstract: ILC2 cells play a role in the pathogenesis of graft versus host disease (GvHD) and idiopathic pneumonia syndrome (IPS), both conditions associated with allogeneic stem cell transplantation. Infusion of IL-33 activated ILC2 cells into patients with ongoing GvHD or IPS, or prior to onset of GvHD or IPS in susceptible patients, substantially ameliorates the disease and improves survival.