Abstract: Methods for designing bivalent compounds which selectively degrade/disrupt EZH2 and compositions and methods of using such degraders/disruptors to treat EZH2-mediated cancer are provided.

Abstract: A genus of dihydropyridine chemical modulators of synaptojanin is disclosed. These modulators are selective inhibitors of synaptojanin 1 and may be used to treat cognitive impairment or traumatic brain injury, including promoting regeneration in cases of traumatic brain injury, or for treating neurodegenerative disorders.

Abstract: Provided herein are antibodies that bind to neuraminidase (NA) of different strains of influenza B virus, host cells for producing such antibodies, and kits comprising such antibodies. Also provided herein are compositions comprising antibodies that bind to NA of different strains of influenza B virus and methods of using such antibodies to diagnose, prevent or treat influenza virus disease.

Abstract: Provided herein are monoclonal antibodies and antigen-binding fragments thereof that bind to the John Cunningham virus. Also provided are pharmaceutical compositions comprising the monoclonal antibodies and antigen-binding fragments thereof, as well as methods of using such monoclonal antibodies and antigen-binding fragments thereof, including methods for the treatment and/or prevention of JCV infections and/or progressive multifocal leukoencephalopathy.

Abstract: The present disclosure is directed towards chimeric glycoproteins wherein the clip region, a core region, a flap region, and a transmembrane and cytoplasmic domain are defined by starting from the amino terminus of the protein, these domains are comprised of the following amino acid residue ranges: clip, 1 through 40 to 60; core, 40 to 60 through 249 to 281; flap, 249 to 281 through 419 to 459; the transmembrane domain is comprised of amino acids 460 through 480, and the remaining amino acids 481 through 525 comprise the cytoplasmic domain; and wherein the clip, core, flap, transmembrane, and cytoplasmic domain comprise a chimeric combination of at least two lyssavirus, wherein the chimeric glycoprotein is advantageously inserted into a rabies-based vaccine vector.

Abstract: Disclosed herein are methods of treatment for an intraocular pressure (IOP)-associated condition in a subject, that include administering to the subject an effective amount of a tissue plasminogen activator (tPA) therapeutic agent. In one embodiment, the IOP-associated condition is glaucoma. The administration of a tPA therapeutic agent can be an extended administration intended to cause a reduction in IOP in the subject for a period of at least one day to a year or more, relative to IOP levels in the subject prior to administration of the tPA therapeutic agent. The tPA therapeutic agent can be, for example, tPA, a tPA derivative, a small molecule direct or indirect tPA agonist, or a gene therapy vector.

Abstract: Disclosed herein are serine threonine kinase (AKT) degradation/disruption compounds including an AKT ligand, a degradation/disruption tag, and a linker, and methods of using such compounds in the treatment of AKT-mediated diseases.

Abstract: The present invention relates to methods for detecting, diagnosing, prognosing, monitoring, and treating a patient with hepatocellular carcinoma. In particular, the invention provides diagnostic markers for the detection and treatment of patients who would benefit from immunotherapy, i.e., patient who would be most responsive to immunotherapy.

Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.

Abstract: The disclosure provides polypeptides that specifically bind to HLA-DQ8 for treating Type 1 Diabetes (TID) and methods using same for reducing autoimmune destruction of pancreatic islet beta cells. In particular, the present disclosure relates to peptides containing at least one D-amino acid that are capable of blocking the presentation of antigenic islet peptides (e.g., lnsB:9-23) by HLA-DQ8, and to their uses, especially as it relates to the prevention and/or treatment of TID.

Abstract: Compositions and methods for diagnosing and treating cancers that have developed from neuronal cells and neuronal cell lines.

Abstract: By hijacking endogenous E3 ligase to degrade protein targets via the ubiquitin-proteasome system, PROTACs (PRoteolysis TArgeting Chimeras) provide a new strategy to inhibit protein targets that were previously regarded as undruggable. The compounds described herein comprise a photolabile group on PROTACs, enabling the degradation of protein targets in a spatiotemporally controlled manner By adding a photolabile caging group on ubiquitin recruiting moieties, light-inducible protein degradation was acheived. These opto-PROTACs display no activity in the dark, while restricted degradation can be induced at a specific time and rate by UVA-irradiation. Accordingly, these compounds provide light-controlled PROTACs and methods of using such compounds.

Abstract: Disclosed herein are controlled release compositions comprising riluzole and the uses thereof.

Abstract: A cranial drill guide includes a housing and a drill guide sleeve assembly pivotally attached to the housing. The drill guide sleeve assembly has at least two degrees of freedom to allow the drill guide sleeve assembly to be moved to a first pivoted position for forming an angled burr hole through a cranial surface. The two degrees of freedom can comprise a pivoting motion of the drill guide sleeve assembly and an axial motion of a portion of the drill guide sleeve assembly.

Abstract: A system and method for determining native cardiac output of a heart while maintaining operation of an intracardiac blood pump includes determining a current drawn by the pump motor, a blood pressure within the ascending aorta, and a change in the blood temperature based on a thermodilution technique. An intracardiac blood pump positioned in the aorta includes at least one sensor for determining a motor current and blood pressure and a thermistor for determining the change in blood temperature after a precise fluid bolus has been introduced into the vasculature. A processor receives current, pressure, and temperature measurements, and calculates a pump flow output and a total cardiac output from which the native cardiac output is calculated. The native cardiac output and other clinically relevant variables derived from the measurements inform decisions related to continued therapeutic care, including increasing or decreasing cardiac assistance provided by the intracardiac pump.

Abstract: The invention relates generally to the THAP1 gene and mutations in this gene, as well as the THAP1 protein and mutations in this protein, that are associated with dystonia. The invention relates to the identification, isolation, cloning and characterization of the DNA sequence corresponding to the wild type and mutant THAP1 genes, as well as isolation and characterization of their transcripts and gene products. The invention further relates to methods and kits useful for detecting mutations in THAP1 that are associated with dystonia, as well as to methods and kits useful for diagnosing dystonia. The present invention also relates to therapies for treating dystonia, including gene therapeutics and protein/antibody based therapeutics.

Abstract: Provided herein are chimeric hemagglutinin (HA) polypeptides and uses thereof for inducing an immune response (e.g., an antibody response) against influenza virus. Also provided herein are methods of generating antibodies to the chimeric HA polypeptides in a subject.

Abstract: The present disclosure relates to methods for the treatment of sarcoidosis. In certain aspects and embodiments, the disclosure provides compositions containing 1,3-propanedisulfonic acid or a pharmaceutically acceptable salt thereof and/or the use of such compositions for the treatment of Sarcoidosis. In another aspect, the disclosure relates to compositions containing 1,3-propanedisulfonic acid or a pharmaceutically acceptable salt thereof plus a second active agent. In yet another aspect, the disclosure relates to kits containing agents useful for the treatment of sarcoidosis.

Abstract: In one aspect, provided herein are mutated influenza virus neuraminidase polypeptides, wherein the mutated influenza virus neuraminidase polypeptides comprise a first cytoplasmic domain, a first transmembrane domain, a first stalk domain, and a first globular head domain of a first neuraminidase of a first influenza virus with an insertion of 15 to 45 or 1 to 50 amino acid residues in the first stalk domain of the first neuraminidase. In another aspect, provided herein is an influenza virus comprising such a mutated influenza virus neuraminidase polypeptide, a genome comprising a nucleotide sequence encoding such a mutated influenza virus neuraminidase polypeptide or both. In another aspect, provided herein is an immunogenic composition comprising such an influenza virus, and optionally an adjuvant.

Abstract: Methods and compositions useful in the prevention and treatment of hair loss. In some embodiments, compositions are applied to hair follicles to inhibit the transition of growth phases. In some embodiments, the composition is a myosin light chain kinase (MLCK) inhibitor alone, or in conjunction with other components. In some embodiments, methods are disclosed for identifying and/or selecting compositions for use in methods of preventing or treating hair loss.