38 results about "Double stranded rna" patented technology

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.

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 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.

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 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 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.

Described herein are synthetic methods for producing sequence-specific RNA oligonucleotides that eliminate impurities produced in prior art methods. In one aspect, a first amplification primer includes one or more deoxyuridine residues, wherein at least one of the one or more deoxyuridine residues is at position −1, −2, −3, −4 or −5 of the promoter region for the single-subunit, DNA-dependent RNA polymerase. The deoxyuridines are excised to provide an amplified functional template DNA which is then used to synthesize RNA which has reduced immunogenic double stranded RNA compared to controls.

The present invention provides methods and systems to identify and quantify double-stranded RNA (dsRNA) in a sample. A capture molecule can be immobilized to a solid surface. The solid surface can then be contacted with a sample including the dsRNA, wherein the capture molecule immobilized on the solid surface specifically binds to the dsRNA. The binding response of the dsRNA to the solid surface can then be measured by biolayer interferometry to detect the dsRNA in the sample. The measured binding response can be compared to a standard curve relating binding response to concentration to determine a concentration of the dsRNA in the sample.

The present invention relates to a novel affinity separation matrix that comprises novel binding ligands for double stranded RNA (dsRNA) and the use of said affinity separation matrix for purifying ribonucleic acid preparations. The present invention further relates to a method of purifying ribonucleic acid preparations and a kit for use in a method of purifying ribonucleic acid preparations, implementing the novel affinity separation matrix. The present disclosure also relates to a method of production of a single-stranded RNA (ssRNA) preparation.

Antibody-oligonucleotide drug conjugates (AODC) targeting CHK1 and / or WEE1 are provided for use in pharmaceutical compositions and methods of treating various cancers. AODCs comprise an antibody, a peptide linker and an oligonucleotide containing GEM moieties. In some embodiments, the oligo comprises a double stranded RNA molecule, which comprises one or more gemcitabine (GEM) moieties replacing certain cytidine nucleotides or other nucleotides. When the sense strand is separated from the antisense strand, one or both of which contain at least one GEM moiety, the sense strand releases its GEM moieties and together with the CHK1-siRNA (containing GEM), exerts a synergistic effect that exceeds the effects of either the released GEM moieties or the CHK1-siRNA-GEM construct alone to silence the CHK1 gene. In some embodiments the RNA molecule of the AODC is an mxRNA containing GEM moieties or an muRNA construct with GEM moieties in one or both antisense strands, targeting both CHK1 and WEE1.

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 compound of formula (I) is contained inside the nucleotide, or at the 5 '-and / or 3'-terminus, for delivery of the double-stranded RNA to extrahepatic tissue, such as the central nervous system or the eye. Oligonucleotides, double-stranded RNAs, vectors, cells, pharmaceutical compositions and kits comprising the compounds of Formula (I) are also provided.

Provided herein double stranded RNA (dsRNA) mi29b mimics and methods of treating fibrotic diseases or disorders with the same.

The present invention relates to a method for the detection of small non-coding RNAs in a sample using sequence-specific structured adapters for the ligation by a double stranded RNA ligase with subsequent cDNA production and quantification. The present invention further relates to a kit for carrying out this method.

Provided herein double stranded RNA (dsRNA) mi29b mimics and methods of treating fibrotic diseases or disorders with the same.

The present invention relates to the field of nucleic acid production, in particular in vitro RNA transcription. More specifically, the present invention relates to a method to reduce formation of double stranded RNA during in vitro transcription, more in particular by the use of particular amounts of Mg during the RNA transcription process. The invention further relates to an in vitro transcribed RNA composition obtainable by the method according to the invention.

The present disclosure provides a double-stranded nucleic acid, compositions, and methods relating thereto. The present disclosure encompasses the recognition that structural elements of double stranded nucleic acid, such as stereochemistry of backbone chiral centers (chiral internucleonic linkages), and / or patterns thereof, specially, double-stranded nucleic acid having one or more chirally enriched phosphorothioate internucleotide linkages can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. The present disclosure also provides methods for treatment of diseases using provided double stranded nucleic acid compositions, for example, in RNA interference.

Provided are TrkA RNAi agents for reducing expression of TrkA, which comprise a sense strand and an antisense strand forming a double stranded RNA (dsRNA), compositions comprising these RNAi agents and methods of their use.

The present invention relates to double-stranded RNA molecules having A to G substitutions, precursor RNA molecules thereof, and their use in modifying cells such as for gene silencing.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Angiopoietin-like 3 (ANGPTL3) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an ANGPTL3 gene and to methods of preventing and treating an ANGPTL3-associated disorder, e.g., a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a solute carrier family member gene, e.g., SLC30A10, or SLC39A8. The invention also relates to methods of using such RNAi agents to inhibit expression of a solute carrier family member gene, e.g., an SLC30A10 gene, or an SLC39A8 gene, and to methods of preventing and treating a solute carrier family member-associated disorder, e.g., a hypermanganesemia.

A method of inhibiting skin cancer by administering to a subject in need thereof a double stranded RNA interference (RNAi) agent comprising at least one of (i) a first double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a CD320 gene wherein the first dsRNA comprises a sense strand and an antisense strand forming a duplex, and (ii) a second dsRNA for inhibiting the expression of a LRP2 gene wherein the second dsRNA comprises a sense strand and an antisense strand forming a duplex, wherein the sense strand of the first dsRNA is at least substantially complementary to the antisense strand of the first dsRNA and the sense strand of the second dsRNA is at least substantially complementary to the antisense strand of the second dsRNA and the use of the RNAi agent as a pharmaceutical composition for the treatment of cancer in subjects in need of treatment.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the apolipoprotein C3 gene (APOC3). The invention also relates to methods of using such RNAi agents to inhibit expression of an APOC3 gene and to methods of preventing and treating an APOC3-associated disorder, e.g., hypertriglyceridemia, non-alcoholic fatty liver disease, non-alcoholic steatohepatitis, polycystic ovary syndrome, kidney disease, obesity, type 2 diabetes mellitus (insulin resistance), hypertension, artherosclerosis and pancreatitis.