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Небесная энциклопедия

Космические корабли и станции, автоматические КА и методы их проектирования, бортовые комплексы управления, системы и средства жизнеобеспечения, особенности технологии производства ракетно-космических систем

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Мониторинг СМИ

Мониторинг СМИ и социальных сетей. Сканирование интернета, новостных сайтов, специализированных контентных площадок на базе мессенджеров. Гибкие настройки фильтров и первоначальных источников.

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Форма поиска

Поддерживает ввод нескольких поисковых фраз (по одной на строку). При поиске обеспечивает поддержку морфологии русского и английского языка
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Применить Всего найдено 56203. Отображено 100.
05-01-2012 дата публикации

Methods for treating neoplasia by inhibiting lactate dehydrogenase and/or nicotinamide phosphoribosyltransferase

Номер: US20120003156A1
Принадлежит: JOHNS HOPKINS UNIVERSITY

The invention provides compositions for the diagnosis or treatment of neoplasias, including lymphomas, leukemias, brain cancers (e. glioblastomas, medulloblastomas), breast cancer, colon cancer, and pancreatic cancer, and methods of use therefor.

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05-01-2012 дата публикации

Compositions and methods for modulating activity of capped small rnas

Номер: US20120004279A1
Принадлежит: Leland Stanford Junior University

Compositions and methods for modulating transcription by RNA polymerases are described.

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05-01-2012 дата публикации

Animal model for parkinson's disease

Номер: US20120005765A1
Принадлежит: St Louis University

Disclosed are methods and compositions for an animal model of Parkinson's disease. In particular, disclosed is the use of antisense compounds to inhibit the expression of ALDH1A1 in the substantia nigra of an animal brain for the purpose of creating an animal that will displays the symptoms of a human with Parkinson's Disease, including various biochemical, histological, and behavioral characteristics. Also disclosed are methods for using the animal model for Parkinson's disease to test potential therapeutic agents for Parkinson's disease.

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19-01-2012 дата публикации

Nucleic acid aptamers

Номер: US20120014875A1

The present invention relates to optimized aptamers and methods of using these aptamers.

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19-01-2012 дата публикации

Novel inhibitors of retroviral reverse transcriptace

Номер: US20120015874A1
Принадлежит: University of Missouri System

Disclosed are nucleic acid molecules, and methods of their use, which have a specific structure including a double helical domain and a G-quadruplex domain physically connected by a linker domain which may be nucleosidic or non-nucleosidic. These aptamers demonstrate potent inhibition of phylogenetically diverse primate lentiviral reverse transcriptases, which effect is largely independent of aptamer sequence provided that the aptamer has the specified structure.

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19-01-2012 дата публикации

RNAi MOLECULE TARGETING THYMIDYLATE SYNTHASE AND APPLICATION THEREOF

Номер: US20120016012A1
Принадлежит: Delta Fly Pharma Inc

This invention provides a novel RNAi molecule that can significantly potentiate antitumor effects of a 5-FU antitumor agent. The RNAi molecule comprises the nucleotide sequence shown in SEQ ID NO: 2. The invention also provides an antitumor agent comprising such RNAi molecule and a 5-FU antitumor agent.

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19-01-2012 дата публикации

Isolated novel nucleic acid and protein molecules from corn and methods of using those molecules to generate transgene plants with enhanced agronomic traits

Номер: US20120017292A1
Принадлежит: MONSANTO TECHNOLOGY LLC

This disclosure provides purified nucleic acids and polypeptides. Also provided are transgenic plants, seeds, and plant cells containing DNA for expression of the proteins that are useful for imparting enhanced agronomic trait(s) to transgenic crop plants, methods of making such plants and methods of making agricultural commodity including seeds and hybrid seeds from such plants.

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26-01-2012 дата публикации

MHC-less cells

Номер: US20120020939A1
Принадлежит: SEARETE LLC

The present disclosure relates to compositions, methods, systems, computer-implemented methods, and computer program products thereof that relate to biological cells for delivery of at least one therapeutic agent to a biological tissue or subject.

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26-01-2012 дата публикации

Short Interfering Ribonucleic Acid (siRNA) for Oral Administration

Номер: US20120022139A1
Принадлежит: NOVARTIS AG

Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.

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02-02-2012 дата публикации

Novel lipids and compositions for the delivery of therapeutics

Номер: US20120027796A1
Принадлежит: Alnylam Pharmaceuticals Inc

The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structures: (Formula (I) or (XXXV)).

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02-02-2012 дата публикации

Short Interfering Ribonucleic Acid (siRNA) for Oral Administration

Номер: US20120029052A1
Принадлежит: NOVARTIS AG

Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.

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09-02-2012 дата публикации

Tyrosine kinase receptor tyro3 as a therapeutic target in the treatment of cancer

Номер: US20120034167A1

The present invention concerns new methods for treating cancer by using TYRO3 inhibitors and methods for identifying new molecules of interest for treating cancer.

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09-02-2012 дата публикации

Marker for Liver-Cancer Diagnosis and Recurrence and Survival Prediction, a Kit Comprising the Same, and Prognosis Prediction in Liver-Cancer Patients Using the Marker

Номер: US20120034235A1

A composition for detecting a marker for the diagnosis or prognosis of liver cancer is disclosed. The composition includes an agent capable of assessing the expression level of UQCRH (ubiquinol-cytochrome c reductase hinge protein). In addition, a kit having the composition, a microarray for the diagnosis of liver cancer using the marker, and a method for detecting the marker, and predicting recurrence following surgery in liver cancer patients are disclosed. The marker is able to contribute to the early diagnosis of liver cancer and prediction of recurrence following surgery and survival of liver cancer patients who have undergone hepatic resection, and also is significant for being a promising therapeutic target for liver cancer.

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09-02-2012 дата публикации

Dual targeting of mir-208 and mir-499 in the treatment of cardiac disorders

Номер: US20120035243A1
Автор: Eric Olson, Eva Van Rooij
Принадлежит: University of Texas System

The present invention provides a method of treating or preventing cardiac disorders in a subject in need thereof by inhibiting the expression or function of both miR-499 and miR-208 in the heart cells of the subject. In particular, specific protocols for administering inhibitors of the two miRNAs that achieve efficient, long-term suppression are disclosed. In addition, the invention provides a method for treating or preventing musculoskeletal disorders in a subject in need thereof by increasing the expression or activity of both miR-208 and miR-499 in skeletal muscle cells of the subject.

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16-02-2012 дата публикации

Ribonucleic Acid Interference Molecules

Номер: US20120040460A1
Принадлежит: International Business Machines Corp

Ribonucleic acid interference molecules are provided. In one aspect of the invention, a method for regulating gene expression comprises the following step. At least one nucleic acid molecule comprising at least one of one or more precursor sequences having SEQ ID NO: 1 through SEQ ID NO: 103,948, each one of the precursor sequences containing one or more mature sequences having SEQ ID NO: 103,949 through SEQ ID NO: 230,447, is used to regulate the expression of one or more genes.

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16-02-2012 дата публикации

Isolation of factors that associate directly or indirectly with chromatin

Номер: US20120040857A1
Принадлежит: General Hospital Corp

Methods for isolating non-coding nucleic acids that are associated with chromatin at a target genomic locus are provided. The methods comprise the steps of obtaining a sample that comprises a target genomic DNA sequence and one or more non-coding nucleic acids associated with that DNA sequence; contacting the sample with at least one oligonucleotide probe that comprises a sequence that is complimentary to and capable of hybridising with at least a portion of the target DNA sequence, wherein the oligonucleotide probe comprises at least one modified nucleotide analogue and wherein the oligonucleotide probe further comprises at least one affinity label; allowing the at least one oligonucleotide probe and the target DNA sequence to hybridise with each other so as to form a probe-target hybrid; isolating the probe-target hybrid from the sample by immobilizing the probe-target hybrid through a molecule that binds to the at least one affinity label; and eluting the one or more non-coding nucleic acids that are associated with the target genomic DNA sequence. Also provided are probes suitable for use in the methods of the invention. The methods and probes of the invention are suited to identification of non-coding RNAs including microRNAs and snoRNAs that are associated with chromatin remodelling.

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16-02-2012 дата публикации

Target substance detection method using aptamer

Номер: US20120040865A1
Автор: So Youn Kim

The present invention relates to a method and kit of detecting a target material using an aptamer, and more particularly to a method and kit for detecting a target material, in which a sample and a second aptamer are added to a first aptamer immobilized on a solid phase so as to form a bond sandwiched between the first aptamer, the target material and the second aptamer, to an FET sensor-based method and kit for detecting a target material, and to an AAO sensor-based method and kit for detecting a target material. The inventive method for a target material using an aptamer can detect even low-molecular-weight materials which were difficult to detect in the prior art, thereby enabling detection of disease-related metabolites, environmental pollutants and food toxins in solutions. In addition, the detection method of the present invention is a direct and simple method and is highly cost-effective, because it uses the aptamer which can be consistently reproduced and can be produced at low costs. Thus, the present invention is very useful.

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23-02-2012 дата публикации

Composition and Methods for the Diagnosis and Treatment of Tumor

Номер: US20120045390A1
Принадлежит: Genentech Inc

The present invention is directed to compositions of matter useful for the diagnosis and treatment of tumor in mammals and to methods of using those compositions of matter for the same.

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23-02-2012 дата публикации

Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family

Номер: US20120046345A1
Принадлежит: OPKO CURNA LLC

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Dystrophin family, in particular, by targeting natural antisense polynucleotides of Dystrophin family. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of DMD family.

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01-03-2012 дата публикации

Splice-region antisense composition and method

Номер: US20120053228A1
Принадлежит: AVI Biopharma Inc

Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5′ end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.

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08-03-2012 дата публикации

Adenoviral va1 pol iii expression system for rnai expression

Номер: US20120058522A1
Принадлежит: CITY OF HOPE

An adenoviral VA1 Pol III expression system for RNAi expression is provided.

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08-03-2012 дата публикации

METHOD OF DIAGNOSING POOR SURVIVAL PROGNOSIS COLON CANCER USING miR-203

Номер: US20120058914A1

The present invention provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the present invention provides diagnostics and prognostics for colon (including colon adenocarcinoma) cancer patients, wherein the methods related to measuring miR levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.

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08-03-2012 дата публикации

Inhibition of map4k4 through rnai

Номер: US20120059046A1
Принадлежит: RXi Pharmaceuticals Corp

RNAi constructs directed to MAP4K4 that demonstrate unexpectedly high gene silencing activities, and uses thereof are disclosed. The blunt-ended constructs have a double-stranded region of 19-49 nucleotides. The constructs have selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. For example, the strands may be modified (e.g., one or both ends of the sense strand is modified by 2′-O-methyl groups), such that the construct is not cleaved by Dicer or other RNAse III, the antisense strand may also be modified by a 2′-O-methyl group at the penultimate 5′-end nucleotide to greatly reduce off-target silencing.

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15-03-2012 дата публикации

Novel Target for Regulating Multiple Sclerosis

Номер: US20120064092A1

Methods are provided for decreasing demyelinating inflammatory disease in a subject by inhibiting the activity of chemokine-like receptor 1 (CMKLR1). Methods are also provided for screening for agents that find use in treating demyelinating inflammatory disease in a subject.

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15-03-2012 дата публикации

Use of the gtpase rab27b to diagnose and treat poor prognosis estrogen-receptor-positive breast cancer

Номер: US20120065147A1
Принадлежит: Individual

The present invention relates to evaluating the prognosis of patients with estrogen receptor-positive breast cancer on the basis of Rab27B expression. The invention further relates to a kit comprising an assay for measuring Rab27B levels in said patients and to the usage of Rab27B as a target to screen for drugs capable of inhibiting or diminishing metastasis of said cancer. Furthermore, the invention discloses compounds which can be used to treat estrogen receptor-positive breast cancer.

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15-03-2012 дата публикации

Rna molecules and therapeutic uses thereof

Номер: US20120065246A1
Автор: Pal Saetrom
Принадлежит: Mina Therapeutics Ltd

The invention relates to double-stranded RNA molecules in which each strand of said molecule possesses: (a) sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof; and (b) sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex; and in which at least one strand of said molecule possesses: (c) a seed region of complementarity to at least one seed site present in a 3′ untranslated region of at least one target mRNA molecule. The invention also relates to an algorithm for the design of a double-stranded RNA molecule in which each strand of said molecule possesses: (a) sufficient complementarity to a target mRNA molecule to facilitate cleavage thereof; and (b) sufficient complementarity to the other strand of the double-stranded RNA molecule so as to form a stable duplex; and in which at least one strand of said molecule possesses: (c) a seed region of complementarity to at least one seed site present in a 3′ untranslated region of at least one target mRNA molecule; wherein said algorithm comprises the steps: (i) input a population of mRNA sequences transcribed from one or more genes of interest; (ii) identify all subsequences of at least 12 nucleotides in length within the population of step (i) which are complementary to another subsequence of at least 12 nucleotides in length in the population; (iii) determine a list of candidate bi-functional double-stranded RNA molecules, said list comprising the double-strand RNA duplexes comprising the two complementary subsequences of step (ii); and (iv) sort the list of candidate double-stranded RNA molecules of step (iii) based on their potential to cause translational suppression.

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15-03-2012 дата публикации

siRNA Targeting Apolipoprotein B (APOB)

Номер: US20120065250A1
Принадлежит: Dharmacon Inc

Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for APOB.

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15-03-2012 дата публикации

Mir-21 promoter driven targeted cancer therapy

Номер: US20120065251A1
Принадлежит: Ramot at Tel Aviv University Ltd

The invention provides a nucleic acid construct comprising a promoter sequence derived from microRNA-21 (miR-21) linked to a nucleic acid sequence encoding an anti-cancer agent, an example of which is a toxin. The constructs of the invention are particularly useful for treating tumors expressing miR-21.

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15-03-2012 дата публикации

Test agent for visceral obesity and use thereof

Номер: US20120066777A1

Disclosed are: a method for detecting (diagnosing) visceral obesity in a subject; a test agent useful for the method; a method for searching for a substance that can be used as an active ingredient for ameliorating visceral obesity; and an ameliorating agent for visceral obesity or a medicinal agent for preventing a metabolic disease developed as a result of the progression of visceral obesity. As the test agent, a polynucleotide which comprises at least 15 nucleotides and can hybridize with a nucleotide sequence for coiled-coil domain containing protein 3 (CCDC3) gene or a nucleotide sequence complementary to the nucleotide sequence under stringent conditions or an antibody capable of recognizing CCDC3 protein is used.

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29-03-2012 дата публикации

Control of Gene Expression Using a Complex of an Oligonucleotide and a Regulatory Peptide

Номер: US20120077270A1
Принадлежит: Imperial College Innovations Ltd

A method for suppressing the expression of a selected gene in a cell, the method comprising introducing into the cell a molecule comprising (1) a nucleic acid binding portion which binds to a site or associated with the selected gene which site is present in a genome and (2) an expression repressor portion, wherein the nucleic acid binding portion comprises an oligonucleotide or oligonucleotide mimic or analogue, and wherein the repressor portion comprises a polypeptide or peptidomimetic. Molecules for use in the methods of the invention are provided. The repressor may be a portion of a histone deacetylase or DNA methylase or polypeptide capable of recruiting a histone deacetylase or DNA methylase.

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29-03-2012 дата публикации

Use of a growth-stimulating protein

Номер: US20120077750A1

This invention relates to the inhibition of a newly discovered growth-stimulating protein in an individual. Further, the invention relates to a method for preventing or treating a cancer, or preventing or treating cancer growth, invasion or metastasis, or preventing or treating other hyperproliferative diseases in an individual, by down regulating the expression of said growth-stimulating protein or by inactivating said protein. Still further, the invention concerns a method for diagnosing cancer or other hyperproliferative diseases in an individual based on said growth-stimulating protein.

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19-04-2012 дата публикации

Devices, Systems and Methods for Improving Memory and/or Cognitive Function Through Brain Delivery of SIRNA

Номер: US20120093916A1
Автор: William F. Kaemmerer
Принадлежит: Kaemmerer William F

The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The present invention also provides valuable small interfering RNA vectors, and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject.

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19-04-2012 дата публикации

Novel lipids and compositions for the delivery of therapeutics

Номер: US20120095075A1
Принадлежит: Alnylam Pharmaceuticals Inc

The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure:

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03-05-2012 дата публикации

Nucleic acid delivery compounds

Номер: US20120107264A1
Принадлежит: Nitto Denko Corp

Polymers including two or more different recurring units are disclosed herein. Also disclosed herein are methods of using such polymers to deliver nucleic acids to a cell.

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03-05-2012 дата публикации

Amphoteric liposomal compositions for cellular delivery of small rna molecules for use in rna interference

Номер: US20120107389A1
Принадлежит: Individual

The present invention provides method and pharmaceutical composition for efficient delivery of siRNA (small interfering ribonucleic acids) into cultured mammalian cells. In addition, the present invention provides methods and compositions for knocking down the expression of a specific target gene by treating cells with the formulations comprising cationic amphiphile, a neutral colipid and a small RNA molecule. We demonstrate that our method delivers siRNA efficaciously into animal cells for the purpose of RNA interference. The area of medical science that is likely to benefit most from the present invention is RNAi therapeutics.

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10-05-2012 дата публикации

Peptide/particle delivery systems

Номер: US20120114759A1
Принадлежит: JOHNS HOPKINS UNIVERSITY

Polymeric nanoparticles, microparticles, and gels for delivering cargo, e.g., a therapeutic agent, such as a peptide, to a target, e.g., a cell, and their use for treating diseases, including angiogenesis-dependent diseases, such as age-related macular degeneration and cancer, are disclosed. Methods for formulating, stabilizing, and administering single peptides or combinations of peptides via polymeric particle and gel delivery systems also are disclosed.

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10-05-2012 дата публикации

Anti-MicroRNA Oligonucleotide Molecules

Номер: US20120115220A1
Принадлежит: ROCKEFELLER UNIVERSITY

The invention relates to isolated anti-microRNA molecules. In another embodiment, the invention relates to an isolated microRNA molecule. In yet another embodiment, the invention provides a method for inhibiting microRNP activity in a cell.

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10-05-2012 дата публикации

Compositions and their uses directed to hepcidin

Номер: US20120115930A1
Принадлежит: ISIS PHARMACEUTICALS INC

Disclosed herein are compounds, compositions and methods for modulating the expression of hepcidin in a cell, tissue or animal or preventing, ameliorating or treating anemia. Also provided are methods for prevention, amelioration or treatment of anemia, and for increasing red blood cell count in an animal. Also provided are methods for the prevention, amelioration and/or treatment of low serum iron levels, low red blood cell count and other clinical endpoints of anemia in an animal. These methods may be achieved by administration of compounds or compositions including antisense compounds targeted to a nucleic acid that expresses hepcidin polypeptide combined with an erythropoiesis stimulating agent.

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10-05-2012 дата публикации

Selective inhibitors of cb2 receptor expression and/or activity for the treatment of obesity and obesity-related disorders

Номер: US20120115931A1
Принадлежит: Individual

The invention relates to a method for treating and/or preventing obesity and/or obesity-related disorders by administering to a subject in need thereof a selective inhibitor of cannabinoid type 2 (CB2) receptor expression.

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10-05-2012 дата публикации

Anti-cancer agent, method for inducing apoptosis of cancer cells, and method for screening for anti-cancer agent

Номер: US20120115935A1
Принадлежит: Individual

Disclosed is an anti-cancer agent which can inhibit the progression of cell cycle in the M phase, unlike taxane anti-cancer agents. The anti-cancer agent comprises a compound capable of inhibiting specifically the expression and/or functions of RBM8A depicted in SEQ ID NO: 1, Magoh depicted in SEQ ID NO: 3 or an isoform thereof. The anti-cancer agent can inhibit the expression and/or functions of RBM8A or Magoh in cancer cells to terminate the cell cycle in the M phase, thereby inhibiting the proliferation of the cells. Therefore, the anti-cancer agent can inhibit the proliferation of cancer cells and induce the apoptosis of cancer cells, leading to the cure of cancer.

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17-05-2012 дата публикации

Nucleic acid ligands to ll37

Номер: US20120121533A1
Автор: George W. Jackson
Принадлежит: Biotex Inc

The present invention is directed to nucleic acid ligands to LL37, methods for producing said nucleic acid ligands, and methods for utilizing said nucleic acid ligands. In one exemplary embodiment, for example, this invention relates to nucleic acid ligands exhibiting high specific binding affinity to LL37 peptides, precursors and/or portions thereof. Further, the nucleic acid ligands may bind competitively with native ligands of LL37 and may also inhibit and/or interfere with LL37 function, such as by binding to LL37.

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17-05-2012 дата публикации

Compositions and methods for immunostimulatory rna oligonucleotides

Номер: US20120121551A1
Принадлежит: Gunther Hartmann

The present invention provides 4-nucleotide (4mer) RNA motifs that confer immunostimulatory activity, in particular, IFN-α-inducing activity to a RNA oligonucleotide. The present invention also provides RNA oligonucleotides, including siRNA, with high or low immunostimulatory activity. The present invention further provides the use of the RNA oligonucleotides of the invention for therapeutic purposes.

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17-05-2012 дата публикации

Treatment of oncostatin m receptor beta mediated heart failure

Номер: US20120121579A1

The present invention relates to an inhibitor of the oncostatin M receptor β or an inhibitor of an activator of the oncostatin M receptor β for use in the treatment and/or prevention of heart failure. The present invention also relates to a method of treating and/or preventing heart failure comprising administering a pharmaceutically effective amount of an inhibitor of the oncostatin M receptor β or an inhibitor of an activator of the oncostatin M receptor β to a subject in need thereof. Further, the present invention also relates to methods of identifying a compound suitable as a lead compound and/or as a medicament for the treatment and/or prevention of heart failure.

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17-05-2012 дата публикации

Small interfering rna delivery

Номер: US20120121689A1
Принадлежит: Lipoxen Technologies Ltd

A liposomal siRNA composition is described. The liposomes are formed of neutral liposome forming components, and the composition comprising additionally sugar. The composition provides reduced expression of target gene, without causing systemic toxicity. The composition is produced by a dehydration-rehydration technique to provide high yields and good control of liposome size.

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17-05-2012 дата публикации

Rna sequence-specific mediators of rna interference

Номер: US20120122111A1
Принадлежит: Individual

The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.

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17-05-2012 дата публикации

METHODS AND COMPOSITIONS FOR REDUCING TARGET GENE EXPRESSION USING COCKTAILS OF siRNAS OR CONSTRUCTS EXPRESSING siRNAS

Номер: US20120122217A1
Принадлежит: APPLIED BIOSYSTEMS LLC

The present invention concerns methods and compositions involving the production or generation of siRNA mixtures or pools capable of triggering RNA-mediated interference (RNAi) in a cell. Compositions of the invention include kits that include reagents for producing or generating siRNA pools. The present invention further concerns methods using polypeptides with RNase III activity for generating siRNA mixtures or pools that effect RNAi, including the generation of a number of RNA molecules to the same target gene.

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24-05-2012 дата публикации

Skin Permeating And Cell Entering (SPACE) Peptides and Methods of Use Thereof

Номер: US20120128756A1
Принадлежит: UNIVERSITY OF CALIFORNIA

The present disclosure provides peptides and peptide compositions, which facilitate the delivery of an active agent or an active agent carrier wherein the compositions are capable of penetrating the stratum corneum (SC) and/or the cellular membranes of viable cells.

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24-05-2012 дата публикации

Lipid compositions

Номер: US20120128760A1
Принадлежит: Alnylam Pharmaceuticals Inc

Disclosed herein are lipid compositions comprising a cationic lipid of formula (I), a neutral lipid, a sterol and a PEG or PEG-modified lipid, wherein formula (I) is (F). Also disclosed are methods of producing the cationic lipid of formula (I).

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24-05-2012 дата публикации

Inhibition of ApoE Cleavage Activity in the Treatment of ApoE-Related Disorders

Номер: US20120129782A1
Принадлежит: J David Gladstone Institutes

The present invention provides methods for treating apoE-related disorders. The methods generally involve administering an effective amount of an agent that inhibits activity of an enzyme that cleaves apoE.

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24-05-2012 дата публикации

dsRNA For Treating Viral Infection

Номер: US20120129913A1
Принадлежит: NOVARTIS AG

The invention relates to double-stranded ribonucleic acids (dsRNAs) targeting gene expression of phosphatidylinositol 4-kinase (PI4K), in particular human phosphatidylinositol 4-kinase, catalytic, beta polypeptide (PIK4CB) or human phosphatidylinositol 4-kinase, catalytic, alpha polypeptide (PIK4CA), and their use for treating infection by positive stranded RNA viruses such as hepatitis C virus (HCV). Each dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PIK4CB or PIK4CA target mRNA. A plurality of such dsRNA may be employed to provide therapeutic benefit. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, and including a delivery modality such as fully encapsulated liposomes or lipid complexes. The invention further includes methods for treating diseases caused by positive stranded RNA virus infection using the pharmaceutical compositions; and methods for inhibiting the propogation of positive stranded RNA viruses in and between cells.

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31-05-2012 дата публикации

Treatment of 'c terminus of hsp70-interacting protein' (chip) related diseases by inhibition of natural antisense transcript to chip

Номер: US20120135941A1
Принадлежит: Curna Inc

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of ‘C terminus of HSP70-Interacting Protein’ (CHIP), in particular, by targeting natural antisense polynucleotides of ‘C terminus of HSP70-Interacting Protein’ (CHIP). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of CHIP.

Подробнее
07-06-2012 дата публикации

Use of DR6 and p75 Antagonists to Promote Survival of Cells of the Nervous System

Номер: US20120141456A1
Принадлежит: BIOGEN IDEC MA INC

The present invention relates to Death Receptor-6 (DR6) proteins which are members of the tumor necrosis factor (TNF) receptor family, and have now been shown to be important for regulating apoptosis in cells of the nervous system. In addition, it has been discovered that p75 is a ligand for DR6. As a result, this invention relates to methods for inhibiting the interaction of DR6 and p75 using DR6 and/or p75 antagonists. In addition, the methods described herein include methods of promoting survival of cells of the nervous system using DR6 antagonists, optionally in combination with p75 antagonists, and methods of treating neurodegenerative conditions by the administration of a DR6 antagonists, optionally in combination with a p75 antagonist.

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07-06-2012 дата публикации

Modified siNA

Номер: US20120142011A1
Принадлежит: QIAGEN GmbH

The present invention pertains to the use of at least one abasic modification within the first 8 nucleotide positions of the 5′ region of the antisense strand of a small interfering nucleic acid (siNA) molecule for reducing off-target effects. Provided are suitable modified siNAs, compositions and methods for producing respective siNAs, as well as kits comprising respective siNAs.

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07-06-2012 дата публикации

Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap

Номер: US20120142610A1
Принадлежит: OPKO CURNA LLC

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of ‘IQ motif containing GTPase activating protein’ (IQGAP), in particular, by targeting natural antisense polynucleotides of ‘IQ motif containing GTPase activating protein’ (IQGAP). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IQGAP.

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07-06-2012 дата публикации

Diagnosis and treatment of adrenocortical tumors using human microrna-483

Номер: US20120142753A1

Disclosed herein are methods of diagnosing and treating a malignant adrenocortical tumor, including adrenocortical carcinoma. In some examples, methods of diagnosing a malignant adrenocortical tumor include detecting expression of at least one microRNA (miR) gene product, such as miR-100, miR-125b, miR-195, miR-483-3p, miR-483-5p and IGF2 mRNA in a sample obtained from the subject with an adrenocortical tumor and comparing expression of at least one of these miR gene products and IGF2 mRNA in the sample obtained from the subject to a control. Altered expression of at least one of the miR gene products and IGF2 mRNA, such as a decrease in miR-100, miR-125b or miR-195 or an increase in miR-483-3p, miR-483-5p, and an increase in IGF2 mRNA, in the sample obtained from the subject compared to the control indicates a malignant adrenocortical tumor.

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07-06-2012 дата публикации

Sphingosine-bound siRNA

Номер: US20120142765A1
Принадлежит: Sylentis SA

The invention relates to novel oligomer analogues and their use in oligonucleotide-based therapies. More specifically, the invention concerns oligonucleotides carrying lipid molecules and their use as potential inhibitors of gene expression.

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07-06-2012 дата публикации

Caspase-8 and skin disease

Номер: US20120144504A1
Принадлежит: Yeda Research and Development Co Ltd

The invention relates to the treatment or prevention of an inflammatory skin disease, disorder or condition, by modulating a protein that is normally regulated by caspase-8 in the skin or by increasing caspase-8 activity or level in the skin. Another aspect of the invention relates to methods for diagnosing an inflammatory skin disease, disorder or condition or a predisposition to develop said disease disorder or condition in an individual. Further aspects of the invention relate to methods for identifying target proteins involved in the course or pathology of an inflammatory skin disease, disorder or condition and to methods of screening a candidate compound for treating said disease, disorder or condition. In particular, the invention relates to inflammatory skin diseases such as atopic dermatitis and psoriasis.

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14-06-2012 дата публикации

Lepidopteran Moth Control Using Double-Stranded RNA Constructs

Номер: US20120148525A1
Принадлежит: US Department of Agriculture USDA

Disclosed is a dsRNA construct that relates to a method to control Lepidopteran moths via double-stranded RNA interference of the PBAN/Pyrokinin gene.

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14-06-2012 дата публикации

Methods and compositions for seamless cloning of nucleic acid molecules

Номер: US20120149069A1
Принадлежит: Life Technologies Corp

The present invention is in the fields of biotechnology and molecular biology. More particularly, the present invention relates to cloning or subcloning one or more nucleic acid molecules comprising one or more type IIs restriction enzyme recognition sites. The present invention also embodies cloning such nucleic acid molecules using recombinational cloning methods such as those employing recombination sites and recombination proteins. The present invention also relates to nucleic acid molecules (including RNA and iRNA), as well as proteins, expressed from host cells produced using the methods of the present invention.

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14-06-2012 дата публикации

Method and System for Monitoring Nutritional Uptake as a Function of Microflora Intestinal Gas Levels

Номер: US20120150153A1
Принадлежит: Kimberly Clark Worldwide Inc

Systems and methods for monitoring nutritional uptake of an individual are disclosed. The method can include monitoring microflora intestinal gas concentration levels associated with a patient and adjusting the volume of nutrient provided by the patient with an artificial feeding device based at least in part on the microflora intestinal gas levels associated with the patient. A microflora intestinal gas sensor can be used to monitor the microflora intestinal gas associated with the patient. The microflora intestinal gas sensor can monitor the microflora intestinal gas in a patient's exhaled breath or in the patient's digestive tract. The microflora intestinal gas sensor be included as part of an enteral feeding system at the distal end or outside end of an enteral feeding tube. Systems and methods for monitoring nutritional uptake of an infant based on microflora intestinal gas levels associated with the infant are also disclosed.

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21-06-2012 дата публикации

Method for Inhibiting Scavenger Receptor-A and Increasing Immune Response to Antigens

Номер: US20120156223A1
Принадлежит: Individual

Provided is a method for enhancing an immune response to a desired antigen in an individual. The method is performed by administering to the individual an agent capable of inhibiting class A macrophage scavenger receptor (SR-A) and optionally administering the desired antigen. Also provided is a method for enhancing an immune response to an antigen by administering to an individual a composition containing antigen presenting cells that are characterized by specifically inhibited SR-A. Substantially purified populations of mammalian dendritic cells characterized by specifically inhibited SR-A are also provided.

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28-06-2012 дата публикации

Spinal muscular atrophy (sma) treatment via targeting of smn2 splice site inhibitory sequences

Номер: US20120165394A1
Принадлежит: University of Massachusetts UMass

The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for “intronic splicing silencer”), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA).

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05-07-2012 дата публикации

Further use of protein kinase n beta

Номер: US20120171220A1
Принадлежит: SILENCE THERAPEUTICS AG

The present invention is related to use of protein kinase N beta or a fragment or derivative thereof as a downstream target of the PI 3-kinase pathway, preferably as a downstream drug target of the PI 3-kinase pathway.

Подробнее
05-07-2012 дата публикации

In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages

Номер: US20120172412A1
Принадлежит: Arrowhead Madison Inc

The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.

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05-07-2012 дата публикации

Process for the identification of compounds for treating cancer

Номер: US20120172581A1

Process for the identification of compounds for treating cancer. The invention relates to a method for identifying candidate compounds for use as therapeutic agents for the treatment of cancer, among those who are able to activate the MDA-5 protein or increase NOXA protein levels and to trigger autophagy. It is based on the fact that activation of dsRNA sensor MDA-5 is able to trigger the destruction of cancer cells by activation both autophagy and apoptosis, autonomously and selectively in tumor cells, without provoking the stabilization of the natural antagonist NOXA, MCL-1. The invention also relates to the use of double-stranded RNAs of the same or similar nature such as polyinosinic-polycytidylic acid (pIC), complexed with carriers such as polyethylenimine polycation (PEI), for the manufacture of medicines for the treatment of cancer.

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26-07-2012 дата публикации

Method for determining the presence and concentration of analytes using a nucleic acid ligand and rare earth elements

Номер: US20120190015A1
Принадлежит: Individual

The present invention relates to methods and an apparatus for determining the presence and concentration of an analyte in a sample and the binding of the analyte to a nucleic acid ligand that include measuring the fluorescence emitted by a rare earth element, i.e., terbium, in the presence of the analyte and the nucleic acid ligand. Specific embodiments include the use of terbium and nucleic acid ligands that specifically bind the mycotoxin ochratoxin. A, to detect and quantify ochratoxin A in, for example, food samples such as grain, wine, or beer. The detection of thrombin using terbium and a thrombin-specific nucleic acid ligand is also disclosed. The present invention also relates to a composition comprising a rare earth element as a cation that facilitates the binding of an analyte to a nucleic acid ligand of the analyte.

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26-07-2012 дата публикации

Treatment of patients after stent implantation or balloon dilatation and drug eluting stents

Номер: US20120190580A1

The present invention relates to a nucleic acid molecule for use in the treatment or preventive treatment of a patient after stent implantation or balloon dilatation, wherein the nucleic acid molecule is selected from (a) a single-stranded nucleic acid molecule comprising or consisting of the sequence of SEQ ID NO: 1, 2, 3 or 4 or a sequence having at least 90% sequence identity to the sequence of SEQ ID NO: 1, 2, 3 or 4; (b) a hairpin RNA, wherein one of the regions forming the double-stranded portion of said hairpin RNA comprises or consists of the sequence of SEQ ID NO: 1, 2, 3 or 4 or a sequence having at least 90% sequence identity to the sequence of SEQ ID NO: 1, 2, 3 or 4; (c) an at least partially double-stranded RNA comprising two separate single strands, wherein a region within one of the strands, said region being located within the double-stranded portion of said double-stranded RNA, comprises or consists of the sequence of SEQ ID NO: 1, 2, 3 or 4 or a sequence having at least 90% sequence identity to the sequence of SEQ ID NO: 1, 2, 3 or 4; (d) a nucleic acid molecule encoding the nucleic acid molecule of (a) or the RNA (b); and (e) a nucleic acid molecule or a two nucleic acid molecules encoding the two separate single strands of the RNA of (c). The present invention also relates to a drug eluting stent comprising the nucleic acid molecule according to the invention.

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16-08-2012 дата публикации

Exon skipping therapy for dysferlinopathies

Номер: US20120208865A1

The present invention relates to methods for restoring the function of a mutated dysferlin comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction. Particularly, the splicing of exon 32 is prevented. The present invention also relates to a method for treating a dysferlinopathy in a patient in need thereof, comprising the step of administering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of one or more exons which encode amino acid sequences that cause said dysfunction. Particularly, the splicing of exon 32 is prevented.

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16-08-2012 дата публикации

Bridged artificial nucleoside and nucleotide

Номер: US20120208991A1
Принадлежит: Osaka University NUC

It is an object of the present invention to provide a novel molecule for antisense therapies which is not susceptible to nuclease degradation in vivo and has a high binding affinity and specificity for the target mRNAs and which can efficiently regulate expression of specific genes. The novel artificial nucleoside of the present invention has an amide bond introduced into a bridge structure of 2′,4′-BNA/LNA. The oligonucleotide containing the 2′,4′-bridged artificial nucleotide has a binding affinity for a single-stranded RNA comparable to known 2′,4′-BNA/LNA and has an increased nuclease resistance over LNA. Particularly, it is expected to be applied to nucleic acid drugs because of its much stronger binding affinity for single-stranded RNAs than S-oligo's affinity

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23-08-2012 дата публикации

Diagnostic and Therapeutic Uses for B Cell Maturation Antigen

Номер: US20120213768A1
Принадлежит: BAYLOR RESEARCH INSTITUTE

Biomarkers and therapies against autoimmune diseases, including systemic lupus erythematosus (SLE) are described herein. The present invention is based on the discovery of B cell maturation antigen (BCMA) and BCMA variant expression on SLE monocytes that can be directly associated with disease activity. The findings of the present invention enable the design of monoclonal antibodies or recombinant proteins that can block BCMA and BCMA variants as well as BCMA-bound APRIL.

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23-08-2012 дата публикации

Methods for Diagnosing Colon Cancer Using MicroRNAs

Номер: US20120214694A1
Принадлежит: Ohio State University

The present invention provides novel methods and compositions for the diagnosis and treatment of solid cancers. The invention also provides methods of identifying inhibitors of tumorigenesis.

Подробнее
23-08-2012 дата публикации

Modulation of factor 7 expression

Номер: US20120214862A1
Принадлежит: ISIS PHARMACEUTICALS INC

Disclosed herein are antisense compounds and methods for decreasing Factor 7 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 7 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 7 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.

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23-08-2012 дата публикации

Modified L-Nucleic Acid

Номер: US20120214868A1
Принадлежит: NOXXON PHARMA AG

A modified L-nucleic acid, containing an L-nucleic acid part conjugated to a non-L-nucleic acid part is described. The conjugate has extended retention time in and demonstrates a delayed elimination from an organism.

Подробнее
30-08-2012 дата публикации

Modified double-stranded polynucleotide

Номер: US20120220649A1
Принадлежит: Daiichi Sankyo Co Ltd

The present invention provides a double-stranded polynucleotide having a sense strand polynucleotide consisting of a nucleotide sequence complementary to a target sequence in a target gene, and an antisense strand polynucleotide having a nucleotide sequence complementary to the sense strand polynucleotide, wherein an aryl or heteroaryl compound is bound to a phosphate group at the 5′-end of the antisense strand polynucleotide.

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30-08-2012 дата публикации

Process for triphosphate oligonucleotide synthesis

Номер: US20120220761A1
Принадлежит: Alnylam Pharmaceuticals Inc

The present invention describes simple, efficient, and enzyme-free method of making oligonucleotides with 5′-triphosphate. This invention presents novel process for synthesizing triphosphate oligonucleotides using a diaryl phosphonate as reagent. The process of the present invention is amenable to large-scale, economic 5′-triphosphate oligonucleotide synthesis.

Подробнее
13-09-2012 дата публикации

Methods and compounds for enhancing anti-cancer therapy

Номер: US20120230991A1
Принадлежит: University of Colorado

The invention provides methods of treating neoplastic disorders in a mammal through the inhibition of Mer and/or AxI receptor tyrosine kinases as well as compounds and compositions useful for inhibiting these kinases in these methods. These treatment methods may be combined with the administration of one or more chemo therapeutic agent(s) to enhance the efficacy or minimize the toxicities of the chemotherapeutic agent(s).

Подробнее
13-09-2012 дата публикации

Compositions and methods for prognosis and treatment of prostate cancer

Номер: US20120232124A1
Принадлежит: Rosetta Genomics Ltd

Described herein are compositions and methods for prognosis and treatment of prostate cancer patients. Specifically the invention relates to microRNA molecules associated with the prognosis of prostate cancer, as well as various nucleic acid molecules relating thereto or derived therefrom.

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13-09-2012 дата публикации

Multi-targets interfering rna molecules and their applications

Номер: US20120232126A1
Принадлежит: Biomics Biotechnologies Co Ltd

This invention relates to interfering RNA (iRNA) molecules and their applications, especially multi-targets iRNA molecules and their applications. The said multi-targets iRNA molecules comprised of a sense strand annealed onto at least one antisense strand, each strand is at least 30 nucleotides in length, the sense or antisense strand has at least two segments, which can target at least two RNAs of different genes, or can target at least two portions of an RNA, and wherein the iRNA does not induce an interferon-response when transfected into a cell. The iRNA molecule can interfere with the translation procedure post-transcription, and the target gene is inhibited or blocked, the iRNA does not induce an interferon-response in vivo. The RNA molecules are the active ingredient in preparation of the drug which can regulate one or many genes function.

Подробнее
13-09-2012 дата публикации

Regulation of neurotransmitter release through anion channels

Номер: US20120232127A1

A novel use of anion channels, preferably Ca 2+ -activated anion channels (CAACs), in regulating release of neurotransmitters from neurons and/or astrocytes is provided. More specifically, CAAC activity regulators, agents for regulating neurotransmitter release comprising such CAAC activity regulators, and methods of screening agents for regulating neurotransmitter release using CAAC as a target.

Подробнее
13-09-2012 дата публикации

Treatment of influenza

Номер: US20120232128A1
Принадлежит: Kyorin Pharmaceutical Co Ltd

The present invention provides a double-stranded RNA which inhibits replication of influenza B viruses by RNA interference, in which the double-stranded RNA comprises an RNA having 19 to 25 nucleotides homologous with a part of an mRNA transcribed from a genomic RNA of the influenza B viruses and an antisense RNA thereof.

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27-09-2012 дата публикации

Treating cancer by modulating mex-3

Номер: US20120244170A1
Принадлежит: Individual

The present invention relates to a method for treating cancer in a subject by modulating MEX-3, by administering to said subject a therapeutically effective amount of a modulator of MEX-3.

Подробнее
27-09-2012 дата публикации

Riboswitch based inducible gene expression platform

Номер: US20120244601A1
Принадлежит: UNIVERSITY OF CALIFORNIA

The present disclosure provides a synthetic translation regulator, as well as gene expression cassettes and gene expression constructs comprising the synthetic translation regulator. The present disclosure further provides genetically modified bacterial host cells comprising a subject synthetic translation regulator; and methods of regulating gene expression in such host cells.

Подробнее
04-10-2012 дата публикации

Microrna as a cancer progression predictor and its use for treating cancer

Номер: US20120255043A1
Принадлежит: Taipei Veterans General Hospital

The present invention is based on the findings that a novel function for miR142-3p in the regulation of Sox2, adenylyl cyclase 9 (AC9), and CD133 expressions, and consequently the overall stemness of recurrent GBM cells as well as CSCs, and that miR142-3p modulated tumor-initiating properties in recurrent GBM. The present invention consequently supports the development of novel miRNA-based strategies for brain tumor treatment.

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04-10-2012 дата публикации

Inducible small rna expression constructs for targeted gene silencing

Номер: US20120255045A1

The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic, particularly mam

Подробнее
11-10-2012 дата публикации

Compositions for Controlling Varroa Mites in Bees

Номер: US20120258646A1

An isolated nucleic acid agent is disclosed comprising a nucleic acid sequence which downregulates expression of a gene product of a Varroa destructor mite. Compositions comprising same and uses thereof are also disclosed.

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11-10-2012 дата публикации

Compositions and Methods of Sphingosine Kinase Inhibitors In Radiation Therapy of Various Cancers

Номер: US20120259150A1
Принадлежит: University of Southern California USC

The present invention relates to Sphingosine kinase inhibitors that are useful for treating various cancers. The invention further relates to compositions and methods of SPK inhibitors, including siRNAs, which specifically block gene expression of SPK and potentiates the effect of radiation in the treatment of various cancers.

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18-10-2012 дата публикации

Modular aptamer-regulated ribozymes

Номер: US20120263691A1

An extensible RNA-based framework for engineering ligand-controlled gene regulatory systems, called ribozyme switches, that exhibit tunable regulation, design modularity, and target specificity is provided. These switch platforms typically contain a sensor domain, comprised of an aptamer sequence, and an actuator domain, comprised of a hammerhead ribozyme sequence. A variety of modes of standardized information transmission between these domains can be employed, and this application demonstrates a mechanism that allows for the reliable and modular assembly of functioning synthetic hammerhead ribozyme switches and regulation of ribozyme activity in response to various effectors. In some embodiments aptamer-regulated cis-acting hammerhead ribozymes are provided.

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18-10-2012 дата публикации

Oligomeric compounds and excipients

Номер: US20120264806A1
Принадлежит: ISIS PHARMACEUTICALS INC

The present invention provides method of optimizing the efficacy and potency of antisense compounds. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipients for improved cellular uptake.

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18-10-2012 дата публикации

RNAi-Mediated Inhibition of Histamine Receptor H1-Related Conditions

Номер: US20120264809A1
Принадлежит: Alcon Research LLC

RNA interference is provided for inhibition of histamine receptor H1 mRNA expression, in particular, for treating patients having an HRH1-related condition or at risk of developing an HRH1-related condition such as allergic conjunctivitis, ocular inflammation, dermatitis, rhinitis, asthma, or allergy.

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18-10-2012 дата публикации

Reversible platelet inhibition

Номер: US20120264815A1
Принадлежит: Duke University

The present invention relates, in general, to receptors and to platelet aggregation and, in particular, to a method of inhibiting platelet aggregation using an aptamer that binds to and inhibits the activity of a receptor, such as glycoprotein IIb/IIIa (gpIIb/IIIa), and to aptamers suitable for use in such a method. The invention also relates to antidotes to antiplatelet agents and to methods of using such antidotes to reverse aptamer-induced platelet inhibition. The invention further relates to von Willebrand Factor (VWF) inhibitors, and antidotes therefore, and to methods of using same.

Подробнее
25-10-2012 дата публикации

Methods of sequencing fluorophore-quencher FRET-aptamers

Номер: US20120270221A1
Принадлежит: PRONUCLEOTEIN BIOTECHNOLOGIES LLC

The present invention describes methods for the production and selecting of single chain (single-stranded) fluorescence resonance energy transfer (“FRET”) DNA or RNA aptamers containing fluorophores (F) and quenchers (Q) at various loci within their structures, such that when its specific matching analyte is bound and the FRET-aptamers are excited by specific wavelengths of light, the fluorescence intensity of the system is modulated (increased or decreased) in proportion to the amount of analyte added. F and Q are covalently linked to nucleotide triphosphates (NTPs), which are incorporated by various nucleic acid polymerases such as Taq polymerase during the polymerase chain reaction (PCR) and then selected by affinity chromatographic, size-exclusion or molecular sieving, and fluorescence techniques. Further separation of related FRET-aptamers can be achieved by ion-pair reverse phase high performance liquid chromatography (HPLC) or other types of chromatography. Finally, FRET-aptamer structures and the specific locations of F and Q within FRET-aptamer structures are determined by digestion with exonucleases and mass spectral nucleotide sequencing analysis. Alternatively, single DNA or RNA intrachain FRET-aptamers can be sequenced and the locations of F and Q within the structure can be determined by nanopore sequencing and the locations of F and Q within the structure can be verified by nucleic acid “combing” coupled to high-powered fluorescence microscopy.

Подробнее
25-10-2012 дата публикации

Antiangiogenic Agent and Method for Inhibition of Angiogenesis

Номер: US20120270922A1
Принадлежит: Osaka University NUC

This invention provides an antiangiogenic agent having a higher treatment effect than those of conventional antiangiogenic agents, and a method for inhibiting angiogenesis using the same. An antiangiogenic agent comprising at least one miRNA type selected from the group consisting of miRNAs, pre-miRNAs, and pri-miRNAs, each having a miRNA activity on VE-cadherin.

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25-10-2012 дата публикации

siRNA Targeting Apoliprotein (APOB)

Номер: US20120270926A1
Принадлежит: Dharmacon Inc

Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for APOB.

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25-10-2012 дата публикации

Methods and compositions for dysferlin exon-skipping

Номер: US20120270930A1
Принадлежит: Leids Universitair Medisch Centrum LUMC

The disclosure provides methods and compositions for inducing exon-skipping in a dysferlin pre-mRNA useful, e.g., in restoring function in a dysferlin deficiency. The disclosure also provides improved methods and compositions for generally inducing exon-skipping in a pre-mRNA.

Подробнее
25-10-2012 дата публикации

Identification of rnai targets and use of rnai for rational therapy of chemotherapy-resistant leukemia and other cancers

Номер: US20120272346A1
Принадлежит: COLD SPRING HARBOR LABORATORY

Provided is a mosaic mouse model for use in determining the potency of an shRNA in vivo for reducing survival of cancer cells of chemotherapy-resistant leukemia. The syngeneic mouse recipient is transplanted with tet-on competent leukemia cells carrying a bicistronic nucleic acid construct comprising a promoter operably linked to a fusion gene associated with chemotherapy-resistant leukemia, and a sequence encoding a reverse tet-transactivator protein, such that both coding sequences are co-expressed from the promoter. Also provided are methods of treating soft tissue cancers.

Подробнее
01-11-2012 дата публикации

Modulation of signal transducer and activator of transcription 3 (stat3)expression

Номер: US20120277284A1
Принадлежит: ISIS PHARMACEUTICALS INC

Disclosed herein are antisense compounds and methods for decreasing STAT3 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate hyperproliferative diseases.

Подробнее
08-11-2012 дата публикации

Functions and targets of let-7 micro rnas

Номер: US20120282696A1
Принадлежит: Individual

The present invention concerns methods and compositions for treating or assessing treatment of diseases related to mis-expression of genes or genetic pathways that can be modulated by let-7. Methods may include evaluating patients for genes or genetic pathways modulated by let-7, and/or using an expression profile to assess the condition of a patient or treating the patient with an appropriate miRNA.

Подробнее
08-11-2012 дата публикации

Inhibitors of Atypical Protein Kinase C and Their Use in Treating Hedgehog Pathway-Dependent Cancers

Номер: US20120283194A1
Принадлежит: Leland Stanford Junior University

Methods and compositions are provided for modulating Hedgehog (Hh) pathway signaling in a cell. Aspects of the methods include methods for inhibiting Hh pathway-promoted cancer proliferation and/or metastasis that is promoted by Hh pathway signaling, methods for treating cancers promoted by Hh pathway signaling, and methods for screening candidate agents for the ability to treat a cancer promoted by Hh pathway signaling. In addition, reagents and kits thereof that find use in practicing the subject methods are provided.

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22-11-2012 дата публикации

Modulation of glucagon receptor expression

Номер: US20120295958A1
Автор: Susan M. Freier
Принадлежит: ISIS PHARMACEUTICALS INC

Compounds, compositions and methods are provided for modulating the expression of glucagon receptor. The compositions comprise oligonucleotides, targeted to nucleic acid encoding glucagon receptor. Methods of using these compounds for modulation of glucagon receptor expression and for diagnosis and treatment of disease associated with expression of glucagon receptor are provided.

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29-11-2012 дата публикации

Novel heparanase splice variant

Номер: US20120301475A1
Принадлежит: Compugen Ltd

The present invention, in at least some aspects, is of splice variants of heparanase, as well as diagnostic kits and methods of use, and therapeutic agents and methods of use based thereon, and antibodies specifically binding thereof.

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