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: 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: 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: 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: 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: 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: 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: 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.

Abstract: The disclosure relates to antigen detection reagents and related methods, systems, and kits. The reagents comprise an antigen-binding molecule conjugated to an inorganic component. In some embodiments, the inorganic component possesses catalytic functionality to provide a detectable signal. In some embodiments, the catalytic inorganic component is or comprises a bimetallic nanoparticle. In other embodiments, the inorganic component is a nanoflowers that provides a physical scaffold onto which the antigen-binding component and a reporter component can be loaded, resulting in augmented antigen-binding and reporting capabilities.

Abstract: Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival.

Abstract: Disclosed herein are novel compositions, methods, and systems for determining whether a subject has, or is at risk of developing, or is at a given stage of a condition afflicting a tissue of interest, or determining the tissue or cell provenance of a biological sample, based on expression level of one or more of the novel miRNA and isomiR sequences disclosed herein. The compositions, methods, and systems described herein can be used to diagnose a disease or disorder, or prognose a given stage and/or progression of the disease or disorder, or determine the identity of the tissue or cell in a sample. In some embodiments, the compositions, methods, and systems described herein can be used to develop a treatment for the disease or disorder. For example, in some embodiments, the novel miRNAs can be used as therapeutics for treatment of a disease or disorder.

Abstract: Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.

Abstract: A substituted pyrimidine piperazine compound and uses thereof, and a pharmaceutical composition containing the compound and uses thereof. Wherein the compound has Formula (I), or a stereoisomer, a geometric isomer, a tautomer, an N-oxide, a hydrate, a solvate, a metabolite, a pharmaceutically acceptable salt or a prodrug thereof. The substituted pyrimidine piperazine compound and the pharmaceutical composition containing the compound can be used to inhibit 5-hydroxytryptamine reuptake and/or activate the 5-HT1A receptors. Also, a method of preparing such compounds and pharmaceutical compositions, and uses thereof in the treatment of central nervous system dysfunction.

Abstract: The present invention provides industrially suitable processes for preparing intermediates in the production of substituted polycyclic pyridone derivatives having a cap-dependent endonuclease inhibitory activity. In the process as shown below, wherein each symbol is as defined in the specification, an optically active substituted tricyclic pyridone derivative of the formula (VII) is obtained in high yield and high enantioselectivity by subjecting a compound of the formula (III) or (VI) to intramolecular cyclization with controlling stereochemistry to obtain a compound of the formula (IV) having a removable functional group on an asymmetric carbon, and then removing the functional group thereof.

Abstract: Aptamers having a G-quadruplex structure that bind specifically to the most commonly used azole-class antifungal drugs, biosensors that comprise those aptamers, and invitro methods for determining the level of one of those drugs utilizing those aptamers or biosensors.

Abstract: Provided is an oligonucleotide conjugate comprising an oligonucleotide and two or more linearly connected asialoglycoprotein receptor-binding molecules attached to the oligonucleotide, wherein the oligonucleotide comprises a locked nucleoside analog having a bridging structure between the 4? and 2? positions, is complementary to a human PCSK9 gene, and has inhibitory activity on the expression of the human PCSK9 gene. The oligonucleotide conjugate of the present invention can be used in the field of pharmaceutical products, in particular, the field of the development and production of therapeutic agents for diseases associated with a high LDL cholesterol level.

Abstract: The invention relates to double stranded oligonucleotide complexes comprising an antisense oligonucleotide (AON) and a complementary sense oligonucleotide (SON), for use in the deamination of a target adenosine in a sense target RNA sequence in a cell by an ADAR enzyme, wherein at least the nucleotide in the AON that is directly opposite the target adenosine in the target RNA sequence does not have a 2?-O-alkyl modification and the SON comprises nucleotides that are at least complementary to all nucleotides in the AON that do not have a 2?-O-alkyl modification. The invention further relates to methods of RNA editing using the AON/SON complexes of the invention.

Abstract: The present invention relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering Antagonist A or another pharmaceutically acceptable salt thereof, optionally in combination with another treatment, to a subject in need thereof. The present invention also relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering an anti-C5 agent (e.g., ARC1905), optionally in combination with another treatment, to a subject in need thereof.

Abstract: Disclosed herein are methods for treating a subject afflicted with a cancer having an activating RET alteration by administering an effective amount of a selective RET inhibitor, e.g., Compound 1 or pharmaceutically acceptable salts thereof, including, e.g., administering an amount of 60 mg to 400 mg of the selective RET inhibitor once daily.