Abstract: Therapeutic agents, compositions, and methods are described for use in the treatment of acute or chronic pain, neuroinflammation, or conditions characterized by acute or chronic pain or neuroinflanmmation. The therapeutic agents comprise a synthetic amphipathic helical peptide capable of acting as a mimetic of apoA-I protein.

Abstract: Metapneumovirus (MPV) F proteins stabilized in a prefusion conformation, nucleic acid molecules and vectors encoding these proteins, and methods of their use and production are disclosed. In several embodiments, the MPV F proteins and/or nucleic acid molecules can be used to generate an immune response to MPV in a subject. In additional embodiments, the therapeutically effective amount of the MPV F ectodomain trimers and/or nucleic acid molecules can be administered to a subject in a method of treating or preventing MPV infection.

Abstract: The disclosure provides methods of treating a patient having primary hyperoxaluria or idiopathic hyperoxaluria comprising administering a therapeutically effective amount of compound of the formula and pharmaceutically acceptable salts, solvates, and hydrates thereof to the patient. The variables, e.g. ring A, n, R, R3, R10, X, Y, and Z are defined herein. These compounds act as lactate dehydrogenase inhibitors and are useful inhibiting the conversion of glyoxylate to oxalate. When administered to a patient having a disease or disorder associated with elevated oxalate levels, such as PH type 1, type 2, or type 3 or idiopathic hyperoxaluria the compounds prevent or substantially reduce the amount and buildup of oxalate the patient's kidneys, bladder, urinary tract and other parts of the patient's body.

Abstract: Disclosed are methods of treating or preventing cancer in a mammal, the method comprising: (a) isolating T cells from a tumor sample from the mammal, wherein the isolated T cells are one or both of exhausted and differentiated, and the isolated T cells have antigenic specificity for a tumor-specific antigen expressed by the tumor sample from the mammal, wherein the tumor-specific antigen is a tumor-specific neoantigen or an antigen with a tumor-specific driver mutation; and optionally expanding the numbers of isolated, tumor antigen-specific T cells; and (b) administering to the mammal (i) the isolated T cells of (a) and (ii) a vaccine which specifically stimulates an immune response against the tumor-specific antigen for which the isolated T cells have antigenic specificity.

Abstract: Disclosed are methods of isolating T cells having antigenic specificity for a mutated amino acid sequence encoded by a cancer-specific mutation, the method comprising: identifying one or more genes in the nucleic acid of a cancer cell of a patient, each gene containing a cancer-specific mutation that encodes a mutated amino acid sequence; inducing autologous APCs of the patient to present the mutated amino acid sequence; co-culturing autologous T cells of the patient with the autologous APCs that present the mutated amino acid sequence; and selecting the autologous T cells. Also disclosed are related methods of preparing a population of cells, populations of cells, pharmaceutical compositions, and methods of treating or preventing cancer.

Abstract: Disclosed are methods of selectively expanding a number of T cells. The methods may comprise: modifying human T cells to express a TCR, wherein the TCR comprises a murine constant region; producing a population of cells comprising a number of human T cells expressing the TCR and a number of human T cells not expressing the TCR; and culturing the population of cells in the presence of (i) irradiated feeder cells, (ii) one or more cytokines, and (iii) an antibody, or an antigen-binding portion thereof, wherein the antibody has antigenic specificity for the murine constant region of the TCR, so as to selectively expand the number of T cells expressing the TCR over the number of T cells not expressing the TCR. Also disclosed are related populations of cells, pharmaceutical compositions, and methods of treating or preventing cancer.

Abstract: A method and treatment for testing efficiency and effectiveness of a near infrared photoimmunotherapy treatment includes injecting an antibody photosensitizer conjugate (APC) into a patient, applying radiation to the patient, thereby causing the APC to release a ligand, which is excreted in the patient's urine, detecting the presence of the ligand with liquid chromatography-mass spectrometry, measuring and quantifying an amount of the ligand present in the patient's urine based on analytical results of the liquid chromatography-mass spectrometry, and determining the effectiveness of the near infrared photo-immunotherapy treatment based on the measured quantified amount of the ligand present in the patient's urine so as to determine an amount of APC remaining in the patient.

Abstract: Polynucleotide expression cassettes comprising synthetic polynucleotides encoding human propionyl-CoA carboxylase alpha (synPCCA) are described herein. Related recombinant expression vectors, recombinant adeno-associated viruses (rAAVs), and compositions are also described. Also described are methods of treating a disease or condition mediated by propionyl-CoA carboxylase, comprising administering to a subject in need thereof a therapeutic amount of any of the polynucleotide expression cassettes, recombinant expression vectors, rAAVs, or compositions.

Abstract: Disclosed are methods of obtaining a cell population enriched for T cells having antigenic specificity for a cancer-specific mutation using in vitro stimulation of memory T cells. Also disclosed are related methods of isolating a T cell receptor (TCR), populations of cells, TCRs or antigen-binding portions thereof, pharmaceutical compositions, and methods of treating or preventing cancer.

Abstract: Disclosed are methods to determine a homeostatic steady-state of a biological entity. Also disclosed are methods to quantify an exchange rate between at least two compartments in a biological system, to use Nuclear Magnetic Resonance (NMR) to characterize physiological water transport, and methods for non-invasively measuring transmembrane exchange rates of endogenous water in a biological system under steady-state or non-steady-state conditions in near-real time.

Abstract: Disclosed is a molecule comprising: (a) a first domain, which comprises a targeting moiety; (b) a second domain, which comprises an albumin binding domain (ABD), (c) a third domain, which comprises a furin cleavage sequence (“FCS”), which FCS is cleavable by furin; and (d) a fourth domain, which comprises an optionally substituted Domain III from Pseudomonas exotoxin A (“PE”). Related nucleic acids, recombinant expression vectors, host cells, populations of cells, pharmaceutical compositions, methods of producing the molecule, methods of treating or preventing cancer in a mammal, and methods of inhibiting the growth of a target cell are also disclosed.

Abstract: Disclosed are methods of preparing thymic emigrant cells in vitro, isolated or purified thymic emigrant cells prepared by the methods, and pharmaceutical compositions comprising the same. Further disclosed are methods of treating or preventing a condition in a mammal comprising administering the thymic emigrant cells or pharmaceutical compositions comprising the same to the mammal.

Abstract: An embodiment of the invention provides chimeric antigen receptor (CAR) amino acid constructs. Nucleic acids, recombinant expression vectors, host cells, populations of cells, and pharmaceutical compositions relating to the CAR constructs are disclosed. Methods of detecting the presence of cancer in a mammal and methods of treating or preventing cancer in a mammal are also disclosed. Methods of making the CAR constructs are disclosed.

Abstract: Methods of identifying a subject with a Filovirus infection are provided herein. In some embodiments, the method comprises contacting a biological sample containing antibodies from the subject with one or more peptides comprising amino acid sequences of selected Filovirus epitopes, detecting the presence or absence of an immune complex of antibodies from the biological sample with the one or more peptides; and wherein the presence of the immune complex identifies the subject as having Filovirus infection and the absence of the immune complex identifies the subject as not having Filovirus infection. Further provided are isolated peptides for use in such methods, as well as a solid support linked to one or more of the disclosed peptides.

Abstract: The present invention relates to the field of virology. More specifically, the present invention provides methods and compositions useful for prevention and treatment of human cytomegalovirus (CMV). In one embodiment, a pharmaceutical composition comprises (a) emetine or a derivative thereof; (b) a human cytomegalovirus (HCMV) drug; and (c) a pharmaceutically acceptable carrier. In certain embodiments, the pharmaceutical composition further comprises an adjuvant. In a specific embodiment, the HCMV drug is ganciclovir. In such embodiments, emetine is present at about 1/10 to about 1/100 the normal dosage for amebiasis.

Abstract: The present disclosure relates generally to compositions and methods for inhibiting dipeptide repeat protein (DPR)-ribosomal RNA (rRNA) interaction. In particular, the present technology relates to administering a therapeutically effective amount of one or more compositions that inhibit DPR-rRNA interaction to a subject diagnosed with, or at risk for DPR-associated pathologies, e.g., amyotrophic lateral sclerosis or frontotemporal dementia.

Abstract: The present disclosure provides vaccine compositions comprising at least one adjuvant and at least one therapeutic factor. The disclosure also provides methods of reducing an immune suppressor cell population in a mammal, methods of argumenting an immune response in a mammal, and methods of treating a diseases in a mammal utilizing the vaccine compositions.

Abstract: Antibodies and antigen binding fragments that specifically bind to gp120 and neutralize HIV-1 are disclosed. Nucleic acids encoding these antibodies, vectors and host cells are also provided. Methods for detecting HIV-1 using these antibodies are disclosed. In addition, the use of these antibodies, antigen binding fragment, nucleic acids and vectors to prevent and/or treat an HIV-1 infection is disclosed.

Abstract: Provided herein are compositions and methods for the viral gene therapy (e.g., AAV-directed gene therapy) of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.

Abstract: An embodiment of the invention provides a chimeric antigen receptor (CAR) comprising an antigen binding domain specific for FLT3, a transmembrane domain, and an intracellular T cell signaling domain. Nucleic acids, recombinant expression vectors, host cells, populations of cells, antibodies, or antigen binding portions thereof, and pharmaceutical compositions relating to the CARs are disclosed. Methods of detecting the presence of a proliferative disorder, e.g., cancer, in a mammal and methods of treating or preventing a proliferative disorder, e.g., cancer, in a mammal are also disclosed.