Abstract: Described herein are methods and compositions related to the modulation of progranulin expression or activity in the brain for the treatment of neurodegenerative diseases.

Abstract: The present disclosure provides a method of reducing neurodegeneration and/or TDP43 associated aggregation, comprising knocking down the expression of Nuclear Paraspeckle Assembly Transcript 1 (NEAT1) or LncRNA NEAT1. Also provided are methods for screening a candidate agent that reduces neurodegeneration and/or TDP43 associated aggregation in a cell, treating or preventing a neurodegenerative disorder, delaying or preventing the onset of a neurodegenerative disorder or reducing a risk for developing a neurodegenerative disorder in a subject and determining whether a subject is suffering from, or at a risk of developing a neurodegenerative disorder, comprising measuring the presence of cytoplasmic NEAT1 in a biological sample, wherein the presence is indicative of the risk of developing a neurodegenerative disorder.

Abstract: The present disclosure relates to antisense oligonucleotides, which target SNCA mRNA (e.g., at an intron exon junction) in a cell, leading to reduced expression of SNCA protein. Reduction of SNCA protein expression is beneficial for the treatment of certain medical disorders, e.g., a neurological disorder.

Abstract: Provided herein are compositions, systems, kits, and methods for treating IL-17a related diseases and conditions using an SBE nucleic acid sequence that binds a SEFIR domain of an ACT1 protein.

Abstract: The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.

Abstract: Methods are provided herein for selectively killing senescent cells and for treating senescence-associated diseases and disorders by administering a senolytic agent. Senescence-associated diseases and disorders treatable by the methods using the senolytic agents described herein include cardiovascular diseases and disorders associated with or caused by arteriosclerosis, such as atherosclerosis; idiopathic pulmonary fibrosis; chronic obstructive pulmonary disease; osteoarthritis; senescence-associated ophthalmic diseases and disorders; and senescence-associated dermatological diseases and disorders.

Abstract: This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.

Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.

Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds.

Abstract: Composition(s), device(s), kit(s), and method(s) for an improved analyte detection sensor(s) comprising at least one scavenger protein and method(s) of preserving the functioning and functional life of the improved analyte detection sensor(s).

Abstract: Methods of identifying a compound, such as a test compound, and applications thereof are provided. For example, methods of identifying a compound that preferentially affects, increases, or decreases a level of association of a macromolecule with one or more target condensates or methods of identifying a compound that preferentially causes a macromolecule to associate or disassociate with one or more target condensates are provided. Additionally, methods of designing and/or identifying and/or making a compound, or portion thereof, with a desired characteristic are provided.

Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the xanthine dehydrogenase (XDH) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an XDH gene and to methods of treating or preventing an XDH-associated disease in a subject.

Abstract: Disclosed is a diagnostic kit for quickly diagnosing a target material with high sensitivity using nanoparticles that absorb infrared light and emit infrared light, in which the nanoparticles are maintained in particle size and have enhanced emission intensity.

Abstract: A novel delivery system for drugs, and especially macromolecules such as proteins or oligonucleotides through biological membranes is provided, and specifically delivery of siRNA. The delivery system comprises conjugation of the macromolecule drug to a moiety that enables effective passage through the membranes. Respectively, novel compounds and pharmaceutical compositions are provided, utilizing said delivery system. In one aspect of the invention, the compounds may be utilized in medical practice, for example, in delivery of siRNA or antisense oligonucleotides across biological membranes for the treatment of medical disorders.

Abstract: Wound healing is a complex homeostatic process in which several distinct types coordinate to repair a physical damage. Failure to close wounds contributes to the pathology of conditions like diabetes mellitus, particularly in the elderly. Presented herein are molecules, pharmaceutical compositions, and methods for applying small RNA oligonucleotide technology to wound healing. Small RNA oligonucleotide approaches as disclosed herein provide a therapeutic strategy for improving both basal and pathological wound healing.

Abstract: Providing a method of estimating the number of microparticles such as microorganisms in a sample, without performing complicated operations. The method comprises counting by constant flow the number of target microorganisms contained in the sample at a predetermined flow rate, sectioning measurement data obtained as a result of the constant flow counting into a predetermined number of sections by a predetermined unit time for a section, counting the number of sections in which microorganisms are detected and the number of sections in which they are not detected, in the predetermined number of sections; and estimating the number of microorganisms in the sample, by a statistical method from the flow rate of the sample in the constant flow counting step, the predetermined number of sections and the predetermined unit time in the sectioning step, and the number of sections in which microorganisms are detected in the counting step.

Abstract: A process for preparing compounds of formula (I), or a salt or solvate thereof, including Brexpiprazole, which process comprises cyclization of a compound of formula (II) or (III), or a salt or solvate thereof. The invention also refers to intermediates of said process.

Abstract: Provided is a material-fixing substrate that does not have to use copper as a catalyst because the substrate-bonding site includes a cyclic alkyne to form a covalent bond with a surface of the substrate, and therefore that can reduce damage to a cell, for example, in a case where a to-be-fixed material is the cell. The material-fixing substrate has a to-be-fixed material fixed thereon via a material-fixing agent. The material-fixing agent includes: a substrate-bonding site that forms a covalent bond with a surface of the substrate and includes at least a cyclic alkyne; a hydrophilic site that is bonded to the substrate-bonding site; a light-responsive site that is bonded to the hydrophilic site and changes the skeleton thereof by irradiation with light; and an attachment site to which the to-be-fixed material is attached.

Abstract: The present invention relates to a method for detection, identification, and/or quantification of one or more microbes, microbial peptides, or compounds of microbial origin, comprising the steps of: (a). contacting an object, a substance, or a sample with a luminescent conjugated oligothiophene (LCO); (b). detecting at least one signal of the luminescent conjugated oligothiophene (LCO) of a); and (c). based on said at least one detected signal in b), determining the presence, identity, and/or quantity of the one or more microbes, microbial peptides, or compounds of microbial origin on said object or in said sample. The present invention further relates to diagnostics and a method of diagnosis of microbes, microbial peptides, or compounds of microbial origin.

Abstract: The present disclosure relates to the field of medicine. In particular, it relates to novel antisense oligonucleotides that prevent or reduce exon 8 skipping in the SLC26A4 gene during pre-mRNA splicing, and their use in the treatment of Pendred Syndrome.