Abstract: The present invention provides a PEG-ACS/M-siRNA nanocomposite application, and method for reducing the histamine content during fishmeal storage thereof. A small interfering ribonucleic acid (siRNA) is designed and prepared according to a histidine decarboxylase gene of Morganella morganii (Morganella morganii subsp. morganii KT), and the histidine decarboxylase gene has a sequence of SEQ ID No: 1. A PEG-ACS/M-siRNA nanocomposite is prepared by using a PEGylated arginine-modified chitosan as a carrier. A PEG-ACS/M-siRNA nanocomposite is added to fishmeal in a certain ratio. The method for reducing the histamine content during fishmeal storage has a significant inhibitory effect on the histamine content during fishmeal storage, and can reduce the histamine content in fishmeal by 49%-53%, which has great significance for the control of biogenic amines in fishmeal in the feed industry.

Abstract: The invention provides methods for diagnosis and monitoring of Rett syndrome and other neurodevelopmental disorders by quantitative analysis of miRNAs in bodily fluids.

Abstract: The present invention provides lipid-based nanoparticles (e.g., liposomes or exosomes) having CD47 on their surface and comprising a therapeutic agent (e.g., a therapeutic protein, an antibody, an inhibitory RNA, and/or a small molecule drug). Furthermore, the present invention provides for use of such lipid-based nanoparticles in therapy.

Abstract: The invention relates to an oligonucleotide that supresses the expression of an allele carrying a dominant mutation that causes hereditary sensory neuropathy type I (HSN1), wherein the suppression takes place through hybridisation of said oligonucleotide to the DNA of said allele or to an RNA transcript of said allele, and which either does not suppress the expression of a wild-type allele not containing the dominant mutation or suppresses the expression of said wild-type allele to a lesser extent than it suppresses the expression of the dominant mutant allele; and also pharmaceutical compositions comprising oligonucleotides of the invention and treatments of HSN1 using such oligonucleotides.

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Kallikrein B, Plasma (Fletcher Factor) 1 (KLKB1) gene, the Factor XII (Hageman Factor (F12) gene, or the Kininogen 1 (KNG1) gene, and methods of using such RNAi agents to inhibit expression of a KLKB1 gene, an F12 gene, and/or a KNG1 gene, and methods of treating subjects having an hereditary angioedema (HAE) and/or a contact activation pathway-associated disorder.

Abstract: The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina 1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina 1.

Abstract: Disclosed herein is a method of promoting sustained survival, sustained regeneration, in a lesioned mature neuron, sustained compensatory outgrowth in a neuron, or combinations thereof. The method comprises contacting the lesioned mature neuron with an effective amount of an inhibitor of PTEN and an effective amount of an inhibitor of SOCS3 to thereby promote survival and/or regeneration and/or compensatory outgrowth of the neuron. Therapeutic methods of treatment of a subject with a neuronal lesion by administration of a therapeutically effective amount of an inhibitor of PTEN and a therapeutically effective amount of an inhibitor of SOCS3, are also disclosed, as are pharmaceutical compositions and devices for use in the methods.

Abstract: This disclosure relates to RNA interference (RNAi) reagents for treatment of hepatitis B virus (HBV) infection, compositions comprising same, and use thereof to treat individuals infected with HBV.

Abstract: A method for reducing neovascularization in an ocular tissue is carried out by contacting the tissue with an inhibitor of endomucin expression or activity.

Abstract: The present disclosure provides, in part, methods of discovering immunotherapy targets in vivo, therapeutic compositions (e.g., shRNA, immunoresponsive cells expressing shRNA and/or a chimeric antigen receptors (CAR)), and methods of use thereof.

Abstract: Method for detecting the presence of nucleases in a sample, characterized in that it comprises the steps of: —incubating the sample to be tested for the presence of nucleases with at least one oligonucleotide linker constituting the substrate for the nuclease to be detected, for a sufficient time to cause degradation of said oligonucleotide linker by the nuclease possibly present in the sample, —adding to the sample, upon incubation, colloidal gold nanoparticles comprising gold nanoparticles functionalized with a first probe oligonucleotide and gold nanoparticles functionalized with a respective second probe oligonucleotide, said first and second probe oligonucleotides being complementary to a respective portion of the nucleotide sequence of the oligonucleotide linker, and—examining the possible colour change of the sample as a result of the addition of said nanoparticles, a colour change of the sample to the colour assumed by the colloidal gold particles when aggregated at a distance less than their size bei

Abstract: The application discloses compositions and methods for preventing and treating diabetic retinopathy. It is disclosed herein that an inhibitor of microRNA-let-7b administered to the eye of a subject in need thereof is useful for preventing and treating several problems associated with diabetic retinopathy and plays a role in vasculature stabilization, increasing retinal thickness, reducing retinal capillary dropout, diminishing microvascular leakage, preventing or treating hyperproliferation of microvascular cells, and stabilizing aberrant neovascularization. The invention includes the use of various kinds of inhibitors of microRNA-let-7b, including, but not limited to, an antagomir of miRNA-let-7b. A useful compound of the invention can be administered into the eye, including intravitreally. A useful antagomir is Dy547-mA(*)mA(*)mCmCmAmCmAmCmAmAmCmCmUmAmCmUmAmCmCmU(*) mC(*)mA (*)(3?-Chl).

Abstract: Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-101 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-128 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-101, pri-miR101 or pre-miR101 to the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-128, pri-miR128 or pre-miR128 to the patient. In embodiments, increased levels of microRNA-101 and/or microRNA-128 cause improvement in one or more symptoms of the seizure disorder.

Abstract: Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

Abstract: Disclosed herein are molecules and pharmaceutical compositions that mediate RNA interference against KRAS. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that mediate RNA interference against KRAS.

Abstract: Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-101 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-128 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-101, pri-miR101 or pre-miR101 to the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-128, pri-miR128 or pre-miR128 to the patient. In embodiments, increased levels of microRNA-101 and/or microRNA-128 cause improvement in one or more symptoms of the seizure disorder.

Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.

Abstract: The invention is directed to one or more antisense polynucleotides and their use in pharmaceutical compositions in a strategy to induce exon skipping in the ?-sarcoglycan gene in patients suffering from Limb-Girdle Muscular Dystrophy-2C (LGM-D2C) or in patients at risk of such a disease. The invention also provides methods of preventing or treating muscular dystrophy. e.g., LGMD2C, by exon skipping in the gamma sarcoglycan gene using antisense polynucleotides. Accordingly, in some aspects the invention provides an isolated antisense oligonucleotide, wherein the oligonucleotide specifically hybridizes to an exon target region of a ?-sarcoglycan RNA. In another aspect, the invention provides a method of inducing exon-skipping of a gamma sarcoglycan RNA, comprising delivering an antisense oligonucleotide or a composition to a cell.

Abstract: Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 11 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 11 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.

Abstract: The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.