74 results about "Double stranded rna" patented technology

The present disclosure is directed to controlling pest infestation by inhibiting one or more biological functions in an invertebrate pest. The disclosure discloses methods and compositions for use in controlling pest infestation by feeding one or more different recombinant double stranded RNA molecules to the pest in order to achieve a reduction in pest infestation through suppression of gene expression. The disclosure also discloses methods and compositions for targeted genome editing in the pest in order to achieve a reduction in pest infestation through disruption of protein activity. The disclosure is also directed to methods for making transgenic plants that express the double stranded RNA molecules and targeted genome editing constructs for use in protecting plants from pest infestation.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, Pemphigus, e.g., Pemphigus vulgaris (PV) and Pemphigus foliaceus (PF), and C3 glomerulopathy.

The present invention relates to modulators, e.g., double stranded RNA (dsRNA) agents, antisense polynucleotide agents, antibodies, guideRNAs that effect ADAR editing, or guideRNAs that effect CRISPR editing, that modulate, e.g., inhibit, the expression and / or activity of inhibin subunit beta E (INHBE). The invention also relates to methods of using such modulators to inhibit expression and / or activity of INHBE and to methods of preventing and treating an INHBE-associated disorder, e.g., metabolic disorder, e.g., metabolic syndrome, in a subject.

The present invention is directed to an anti-fungal composition including a plurality of particles including: at least one dsRNA molecule including a nucleic acid sequence complementary to at least one transcript of at least one essential gene of a fungus, and methods of using same.

The present disclosure relates generally to antibodies and antigen-binding fragments that bind to double stranded RNA (dsRNA) and technologies for detection and / or quantification of dsRNA.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a complement component C3 (C3) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), C3 glomerulonephritis, or systemic lupus erythmatosis.

Provided are compositions, systems and methods for removal of double stranded RNA from a mixture utilizing affinity agents having high binding affinity and selectivity for double stranded RNA over single stranded RNA.

Embodiments of the invention include a method of purifying circular RNA (circRNA) from a solution. The method an include steps of (a) incubating the solution at about 55°C, (b) cellulose chromatography, (c) desalting and purifying RNA reactions with magnetic carboxylic acid beads, (d) ethanol precipitation to remove double stranded RNA byproducts and concentrated RNA, (e) poly-A polymerase treatment, (f) desalting and purifying RNA reactions with magnetic carboxylic acid beads to remove poly A reaction components, (g) RNase R treatment, and (h) desalting and purifying the solution to yield purified circRNA. In aspects, the method removes, among other contaminates, in vitro transcribed circular RNA from linear and doubled stranded RNA.

The present disclosure is directed to controlling pest infestation by inhibiting one or more biological functions in an invertebrate pest. The disclosure discloses methods and compositions for use in controlling pest infestation by feeding one or more different recombinant double stranded RNA molecules to the pest in order to achieve a reduction in pest infestation through suppression of gene expression. The disclosure also discloses methods and compositions for targeted genome editing in the pest in order to achieve a reduction in pest infestation through disruption of protein activity. The disclosure is also directed to methods for making transgenic plants that express the double stranded RNA molecules and targeted genome editing constructs for use in protecting plants from pest infestation.

To provide double stranded RNA and methods of use thereof for Machado Joseph disease (MJD).SOLUTION: The disclosure uses RNA silencing technology, e.g., RNA interference, against exonic single nucleotide polymorphism (SNP) in ataxin-3 gene encoding dominant gain-of-function mutant ataxin-3 protein. For that purpose, provided is a highly-target specific gene silencing RNA having an anti-sense sequence that is complementary to a SNP that is in linkage disequilibrium with the disease-causing expansion. Furthermore, the disclosure provides a selected adeno-associated viral vector, in particular serotype 9 (AAV9) as a gene delivery vector, upon which the double stranded RNA can be delivered into the central nervous system (CNS).SELECTED DRAWING: None

This disclosure relates to a nucleic acid comprising a double stranded RNA molecule comprising sense and antisense strands and further comprising a single stranded DNA molecule covalently linked to at least the 5′ end of either the sense or antisense RNA part of the molecule wherein the double stranded inhibitory RNA targets genes associated with cardiovascular disease in the treatment hypercholesterolemia and diseases associated with hypercholesterolemia such as cardiovascular disease.

The present invention relates to mixtures comprising single-stranded (ssRNA) and / or double stranded RNA (dsRNA), compositions comprising these mixtures and methods for the control of phytopathogenic fungi of the genus Erysiphe or Botrytis using these mixtures or compositions. The invention relates further to methods to protect plants producing one or more of these single stranded and / or double-stranded RNAs against phytopathogenic fungi of the genus Erysiphe or Botrytis.

Post-transcriptionally chemically modified double strand RNAs (MdsRNAs) and post-transcriptionally chemically modified RNAs (MRNAs) having a high molecular weight polyalkyloxy modification at the 2′-OH position are described. This modification allows for greater bioavailability of the compound, better stability of the compound, and allows for greater stability against nucleases. The MdsRNAs and MRNAs can be economically produced in a readily scalable manner. Compositions of MdsRNAs and MRNAs having chemically modified nucleotides are described, such that the MdsRNAs and MRNAs are modified to contain high molecular weight polyalkyloxy polymers. Synthetic methods for efficiently making these MdsRNAs and MRNAs and methods of using MdsRNAs and MRNAs are described.

The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.

The disclosure relates to isolated oligonucleotides comprising duplex regions targeting Proprotein convertase subtilisin kexin 9 (PCSK9), and delivery systems, kits and compositions comprising same, and methods of using same for inhibiting or downregulating PCSK9.

Provided herein are engineered guides configured, upon hybridization to target RNA molecules, to form double stranded RNA substrates comprising (i) a region comprising at least one structural feature; and (ii) a first internal loop and a second internal loop, wherein the double stranded RNA substrates recruit RNA editing entities and facilitate chemical modifications of base nucleotides in the target RNA molecules. Also provided herein are compositions, vectors, and cells comprising the engineered guides disclosed herein. Also provided herein are methods of introducing the engineered guides described herein into cells and methods of treating a disease or condition in a subject in need thereof comprising administering to the subject the engineered guides, polynucleotides encoding the engineered guides, delivery vehicles comprising such engineered guides or such polynucleotides, or pharmaceutical compositions comprising any one of these as described herein.

One aspect of this disclosure is directed to a method for treating a cancer in a subject in need thereof by administering to the subject at least a first compound and a second compound in any order together or separately. The first compound is an effective amount of a checkpoint inhibitor optionally with at least one pharmaceutically acceptable carrier. The second compound is an effective amount of an Therapeutic Double Stranded RNA (tdsRNA) optionally with at least one pharmaceutically acceptable carrier. The compounds can be administered together or separately. Compositions for the practice of the method are also described.

The present disclosure provides compositions and methods for using accessory proteins to increase the yield of full-length RNA, to decrease the amount of double stranded RNA, and to increase the efficiency of RNA produced by in vitro transcription.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and to methods of preventing and treating an PNPLA3-associated disorder, e.g., Nonalcoholic Fatty Liver Disease (NAFLD).

The present disclosure is directed to controlling pest infestation by inhibiting one or more biological functions in an invertebrate pest. The disclosure discloses methods and compositions for use in controlling pest infestation by feeding one or more different recombinant double stranded RNA molecules to the pest in order to achieve a reduction in pest infestation through suppression of gene expression. The disclosure also discloses methods and compositions for targeted genome editing in the pest in order to achieve a reduction in pest infestation through disruption of protein activity. The disclosure is also directed to methods for making transgenic plants that express the double stranded RNA molecules and targeted genome editing constructs for use in protecting plants from pest infestation.

The technology described herein relates to 5 '-modified nucleosides, nucleotides, oligonucleotides and double stranded RNAs, such as siRNAs, as well as kits comprising them and their use for inhibition of target genes.

The present invention provides a double-stranded RNA having a nucleotide analogue. The double stranded RNAs of the present invention exhibit reduced off-target toxicity.

The present disclosure relates to methods of treating, preventing, or modulating cardiovascular disease in a patient in need thereof using a double stranded RNA agent that reduces expression of Angiotensin (AGT).

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the AGT gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an AGT gene and to methods of preventing and treating an AGT-associated disorder, e.g., high blood pressure.

The present invention relates to new double stranded RNA (dsRNA) structures and their use in gene silencing.

Compounds of Formula (I), or pharmaceutically acceptable salts, tautomers, or stereoisomers thereof, are provided. The compounds of formula (I) are contained inside oligonucleotides, or at the 5 '-and / or 3'-terminus, for delivery of double-stranded RNA to extrahepatic tissue, such as the eye. The invention also relates to oligonucleotides, double-stranded RNAs, vectors, cells, pharmaceutical compositions and kits comprising the compounds of formula (I).

The invention discloses a nasal delivery double-chain RNA / Mn < 2 + > nano-drug as well as a preparation method and application thereof in immunotherapy. And adding the polymer into a solution containing double-stranded RNA and manganese salt, and self-assembling to form the nasal delivery double-stranded RNA / Mn < 2 + > nano-drug. The invention provides a new technical scheme for the barrier of the BBB. The nano delivery system has the advantages in the aspects of overcoming the BBB and improving the in-vivo biological distribution of the drug, and the surface of the nano carrier is modified with a ligand targeted to an endothelial cell overexpression receptor, such as ApoE peptide and the like, so that the drug can be delivered to penetrate through the BBB; a non-invasive nasal-brain delivery strategy becomes a new scheme for improving drug availability and reducing toxic and side effects, the drug can bypass BBB and rapidly enter brain parenchyma through olfactory bulb and trigeminal nerve conduction pathways, and an immune drug delivered by the nasal cavity can rapidly enter nasopharynx-related lymphatic tissue (NALT) and is transferred to CLN through a lymphatic network around the nasal cavity, so that the immune drug can be rapidly delivered to the nasopharynx-related lymphatic tissue (NALT). Further, a potent local immune response aiming at the in-situ GBM is induced.

Disclosed are double stranded RNA molecules that are toxic to aphids including those that are resistant to pyrethroid insecticides. In particular, interfering RNA molecules capable of interfering with a voltage-gated sodium channel subunit h1 (vgsc-h1) target gene and that are toxic to aphids are provided. Further, methods of making and using the interfering RNA, for example in transgenic plants or as the active ingredient in insecticidal compositions, to confer protection from aphid damage are disclosed.

The present disclosure provides double-stranded RNAs. The present disclosure provides a recombinant nucleic acid comprising: a) a double-stranded RNA of the present disclosure; and b) a microRNA scaffold; the present disclosure also provides a recombinant expression vector comprising a nucleotide sequence encoding such a recombinant nucleic acid. The present disclosure provides a recombinant expression vector encoding a recombinant nucleic acid of the present disclosure. The present disclosure provides compositions and delivery vehicles comprising a double stranded RNA, a recombinant nucleic acid, or a recombinant expression vector of the present disclosure. The present disclosure provides methods for selectively reducing translation and / or accumulation of a disease-associated GGGGCC repeat-containing RNA. The present disclosure provides methods for treating GGGGCC repeat expansion-associated diseases.

The present disclosure provides double-stranded RNAs. The present disclosure provides a recombinant nucleic acid comprising: a) a double-stranded RNA of the present disclosure; and b) a microRNA scaffold; the present disclosure also provides a recombinant expression vector comprising a nucleotide sequence encoding such a recombinant nucleic acid. The present disclosure provides a recombinant expression vector encoding a recombinant nucleic acid of the present disclosure. The present disclosure provides compositions and delivery vehicles comprising a double stranded RNA, a recombinant nucleic acid, or a recombinant expression vector of the present disclosure. The present disclosure provides methods for selectively reducing translation and / or accumulation of a disease-associated GGGGCC repeat-containing RNA. The present disclosure provides methods for treating GGGGCC repeat expansion-associated diseases.