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05-01-2012 дата публикации

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

Номер: US20120003156A1

Автор: Chi V. Dang, Quy H. Le, Ramani Dinavahi

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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Номер записи: 1

19-01-2012 дата публикации

Nucleic acid aptamers

Номер: US20120014875A1

Автор: Anton P. Mccaffrey, James O. McNamara, Paloma H. Giangrande

Принадлежит: University of Iowa Research Foundation UIRF

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

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Номер записи: 2

19-01-2012 дата публикации

RNAi MOLECULE TARGETING THYMIDYLATE SYNTHASE AND APPLICATION THEREOF

Номер: US20120016012A1

Автор: Cheng Long HUANG, Hiromi Wada

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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Номер записи: 3

26-01-2012 дата публикации

Folate targeting of nucleotides

Номер: US20120022245A1

Автор: Christopher Paul Leamon, Iontcho Radoslavov Vlahov, Longwu Qi, Mini Thomas, Paul Joseph Kleindl, Philip Stewart Low

Принадлежит: Endocyte Inc, PURDUE RESEARCH FOUNDATION

The present invention relates to compounds, compositions, kits, and methods of use in targeting nucleotides, such as siRNA's, to cancer cells or to immune system cells involved in inflammation. More particularly, the invention is directed to receptor binding ligand-nucleotide delivery conjugates for use in specifically targeting the conjugates to cancer cells or to immune system cells, methods of treatment with these conjugates, methods of preparation of these conjugates, and methods of reducing the expression of a gene in vitro or in vivo with the conjugates described herein.

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Номер записи: 4

02-02-2012 дата публикации

Novel lipids and compositions for the delivery of therapeutics

Номер: US20120027796A1

Автор: David Butler, Jayaprakash K. Narayanannair, Kallanthottathil G. Rajeev, Laxmab Eltepu, Muthiah Manoharan, Muthusamy Jayaraman

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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Номер записи: 5

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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Номер записи: 6

16-02-2012 дата публикации

Ribonucleic Acid Interference Molecules

Номер: US20120040460A1

Автор: Isidore Rigoutsos, Kevin Charles Miranda, Tien Huynh

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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Номер записи: 7

16-02-2012 дата публикации

Isolation of factors that associate directly or indirectly with chromatin

Номер: US20120040857A1

Автор: Jerome Dejardin, MATTHEW SIMON, Robert Kingston

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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Номер записи: 8

16-02-2012 дата публикации

Target substance detection method using aptamer

Номер: US20120040865A1

Автор: So Youn Kim

Принадлежит: Industry Academic Cooperation Foundation of Dongguk University

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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Номер записи: 9

01-03-2012 дата публикации

Splice-region antisense composition and method

Номер: US20120053228A1

Автор: Patrick L. Iversen, Robert Hudziak

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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Номер записи: 10

01-03-2012 дата публикации

Cationic poly (amino acids) and uses thereof

Номер: US20120053295A1

Автор: Hyunjin Kim, Kanjiro Miyata, Kazunori Kataoka, Nobuhiro Nishiyama, Shourong Wu, Takehiko Ishii

Принадлежит: University of Tokyo NUC

The present invention provides an efficient delivery system for a nucleic acid, more specifically, a cationic poly(amino acid) that has a side chain having a plurality of different amine functional groups in a moiety including a cationic group and that has a hydrophobic group introduced into part of the side chain, and a polyion complex (PIC) of the poly(amino acid) and an oligo- or polynucleotide.

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Номер записи: 11

08-03-2012 дата публикации

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

Номер: US20120058914A1

Автор: Aaron J. Schetter, Carlo M. Croce, Curtis C. Harris

Принадлежит: Ohio State University Research Foundation, US Department of Health and Human Services

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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Номер записи: 12

08-03-2012 дата публикации

Inhibition of map4k4 through rnai

Номер: US20120059046A1

Автор: Anastasia Khvorova, Joanne Kamens, Tod M Woolf, William Salomon

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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Номер записи: 13

15-03-2012 дата публикации

siRNA Targeting Apolipoprotein B (APOB)

Номер: US20120065250A1

Автор: Anastasia Khvorova, Angela Reynolds, Devin Leake, Stephen Scaringe, Steven Read, William Marshall

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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Номер записи: 14

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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Номер записи: 15

19-04-2012 дата публикации

Novel lipids and compositions for the delivery of therapeutics

Номер: US20120095075A1

Автор: David Butler, Kallanthottathil G. Rajeev, Laxman Eltepu, Muthiah Manoharan, Muthusamy Jayaraman, Narayanannair K. Jayaprakash

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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Номер записи: 16

03-05-2012 дата публикации

Nucleic acid delivery compounds

Номер: US20120107264A1

Автор: Akinobu Soma, Chen-Chang Lee, Fu Chen, Jian Liu, Julia Zhu, Keisaku Okada, Lei Yu, Nianchun Ma, Padmanabh Chivukula, Sang Van

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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Номер записи: 17

03-05-2012 дата публикации

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

Номер: US20120107389A1

Автор: Arabinda Chaudhuri, Jayanta Bhattacharyya

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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Номер записи: 18

10-05-2012 дата публикации

Peptide/particle delivery systems

Номер: US20120114759A1

Автор: Aleksander S. Popel, Joel Chaim Sunshine, Jordan Jamieson Green, Kristen Lynn Kozielski, Ron B. Shmueli, Stephany Yi Tzeng

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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Номер записи: 19

17-05-2012 дата публикации

Compositions and methods for immunostimulatory rna oligonucleotides

Номер: US20120121551A1

Автор: Gunther Hartmann, Veit Hornung

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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Номер записи: 20

17-05-2012 дата публикации

Small interfering rna delivery

Номер: US20120121689A1

Автор: Andrew David Bacon, Gregory Gregoriadis, Peter Laing

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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Номер записи: 21

24-05-2012 дата публикации

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

Номер: US20120128756A1

Автор: Samir M. Mitragotri, Tracy Hsu

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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Номер записи: 22

07-06-2012 дата публикации

Modified siNA

Номер: US20120142011A1

Автор: Alexander Azzawi, Eric fat Lader, Jie Kang, Peter Hahn, Wolfgang Bielke

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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Номер записи: 23

07-06-2012 дата публикации

Sphingosine-bound siRNA

Номер: US20120142765A1

Автор: Ana Isabel JIMÉNEZ, Anna Avino, Clara Caminal, Gema Panizo, Ramon Eritja, Santiago Grijalvo, Tamara MARTÍNEZ

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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Номер записи: 24

28-06-2012 дата публикации

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

Номер: US20120165394A1

Автор: Elliot J. Androphy, Natalia N. Singh, Nirmal K. Singh, Ravindra N. Singh

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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Номер записи: 25

05-07-2012 дата публикации

Further use of protein kinase n beta

Номер: US20120171220A1

Автор: Anke Klippel-Giese, Jörg Kaufmann

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.

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Номер записи: 26

05-07-2012 дата публикации

In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages

Номер: US20120172412A1

Автор: Andrei V. Blokhin, Darren H. Wakefield, David B. Rozema, David L. Lewis, Eric A. Kitas, Guenther Ott, Hans Martin Mueller, Ingo Roehl, Jeffrey C. Carlson, Jon A. Wolff, Jonathan D. Benson, Kerstin Jahn-Hofmann, Peter Mohr, Philipp Hadwiger, Torsten Hoffmann

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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Номер записи: 27

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

Автор: Gregory Allen Penner, Jorge Andres Cruz-Aguado

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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Номер записи: 28

16-08-2012 дата публикации

Exon skipping therapy for dysferlinopathies

Номер: US20120208865A1

Автор: Luis Garcia, Marc Bartoli, Martin Krahn, Nicolas Levy

Принадлежит: Institut National de la Sante et de la Recherche Medicale INSERM, Universite de la Mediterranee Aix Marseille II

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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Номер записи: 29

16-08-2012 дата публикации

Bridged artificial nucleoside and nucleotide

Номер: US20120208991A1

Автор: Aiko Yahara, Masaru Nishida, Satoshi Obika, Tetsuya Kodama, Yoshiyuki Hari

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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Номер записи: 30

23-08-2012 дата публикации

Methods for Diagnosing Colon Cancer Using MicroRNAs

Номер: US20120214694A1

Автор: Carlo M. Croce, George A. Calin, Stefano Volinia

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.

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Номер записи: 31

23-08-2012 дата публикации

Modified L-Nucleic Acid

Номер: US20120214868A1

Автор: Bernd Eschgfäller, Christian Lange, Sven Klussmann

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.

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Номер записи: 32

13-09-2012 дата публикации

Compositions and methods for prognosis and treatment of prostate cancer

Номер: US20120232124A1

Автор: Ayelet Chajut, Shai Rosenwald

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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Номер записи: 33

04-10-2012 дата публикации

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

Номер: US20120255043A1

Автор: Guang-Yuh Chiou, Shih-Hwa Chiou

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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Номер записи: 34

18-10-2012 дата публикации

Oligomeric compounds and excipients

Номер: US20120264806A1

Автор: Andrew M. Siwkowski, C. Frank Bennett, Eric E. Swayze, Richard S. Geary

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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Номер записи: 35

25-10-2012 дата публикации

Methods of sequencing fluorophore-quencher FRET-aptamers

Номер: US20120270221A1

Автор: John G. Bruno, Joseph Chanpong

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.

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Номер записи: 36

25-10-2012 дата публикации

siRNA Targeting Apoliprotein (APOB)

Номер: US20120270926A1

Автор: Anastasia Khvorova, Angela Reynolds, Devin Leake, Stephen Scaringe, Steven Read, William Marshall

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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Номер записи: 37

25-10-2012 дата публикации

Methods and compositions for dysferlin exon-skipping

Номер: US20120270930A1

Автор: Annemieke Aartsma-Rus, Garrit-Jan B. van Ommen, Isabella Houweling-Gazzoli, Johannes T. den Dunnen, Silvère Maria VAN DER MAAREL

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.

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Номер записи: 38

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

Автор: Anthony Mazurek, Bruce Stillman, Christopher Vakoc, Johannes Ekkehart Zuber, Katherine McJunkin, Scott W. Lowe

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.

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Номер записи: 39

01-11-2012 дата публикации

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

Номер: US20120277284A1

Автор: Eric E. Swayze, Robert A. MacLeod, Susan M. Freier, Youngsoo Kim

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.

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Номер записи: 40

29-11-2012 дата публикации

Conjugates, particles, compositions, and related methods

Номер: US20120302622A1

Автор: Donald E. Bergstrom, Jungyeon Hwang, Oliver S. Fetzer, Patrick Lim Soo, Pei-Sze Ng, Scott Eliasof, Sonke Svenson

Принадлежит: Cerulean Pharma Inc

Particles and conjugates for delivering nucleic acid agents. Compositions containing the particles, the conjugates, or both. Methods of using the particles, the conjugates, and the compositions.

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Номер записи: 41

27-12-2012 дата публикации

Compositions and methods for vaccinating against hsv-2

Номер: US20120328656A1

Автор: Adrian Vilalta, David M. Koelle, Lichun Dong, Michal Margalith

Принадлежит: UNIVERSITY OF WASHINGTON, Vical Inc

This invention relates to a method for systemic immune activation which is effective for eliciting both a systemic, non-antigen specific immune response and a strong antigen-specific immune response in a mammal. The method is particularly effective for protecting a mammal from herpes simplex virus. Also disclosed are therapeutic compositions useful in such a method.

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Номер записи: 42

27-12-2012 дата публикации

Pharmaceutical Composition Comprising miRNA-100 And Its Use in the Modulation Of Blood Vessel Growth and Endothelial Inflammation

Номер: US20120328686A1

Автор: Christoph Bode, Felix Hans, Franziska Bluhm, Martin Moser, Sebastian Grundmann

Принадлежит: Individual

Disclosed is a pharmaceutical composition comprising a miRNA-100 molecule or an antagomir thereof or a variant thereof for use as a medicament for the positive or negative modulation of blood vessel growth and vascular inflammation wherein the miRNA-100 or the miRNA antagomir has a homology of at least 85% to any of SEQ ID NO:1, 2, 3 and/or 9.

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Номер записи: 43

27-12-2012 дата публикации

Therapeutic agent for fibroid lung

Номер: US20120328694A1

Автор: Rishu Takimoto, Yoshiro Niitsu

Принадлежит: Nitto Denko Corp

Disclosed are: a substance transfer carrier to an extracellular matrix-producing cell in the lung, which comprises a retinoid; a therapeutic agent for fibroid lung, which utilized the carrier; and a preparation kit of the therapeutic agent.

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Номер записи: 44

03-01-2013 дата публикации

Two-way, portable riboswitch mediated gene expression control device

Номер: US20130004980A1

Автор: Jian-Dong Huang, Ye Jin

Принадлежит: University of Hong Kong HKU

A regulatable gene expression construct comprising a nucleic acid molecule comprising a two-way riboswitch operably linked to a target sequence. Also provided is a library screening strategy for efficient creation of target-specific riboswitches. A theophylline-repressible and IPTG-inducible riboswitch device achieves portable control of gene expression control in a ‘two-way’ manner. The default state of target genes is ON; the targets are switched off by adding theophylline, and switched back to the ON-state by adding IPTG without changing growth medium. The riboswitch device regulates gene expression in a portable, adjustable, and two-way manner with a variety of scientific and biotechnological applications.

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Номер записи: 45

10-01-2013 дата публикации

Compositions And Methods For Inhibiting Expression Of GSK-3 Genes

Номер: US20130012572A1

Автор: Dinah Wen-Yee Sah, Gregory Hinkle

Принадлежит: Individual

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting Glycogen Synthase Kinase-3 (GSK-3), and methods of using the dsRNA to inhibit expression of GSK-3.

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Номер записи: 46

07-02-2013 дата публикации

Modulating xrn1

Номер: US20130034543A1

Автор: Helge Grosshans, Saibal Chatterjee

Принадлежит: Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute for Biomedical Research

The present invention relates to a method for modulating miRNA, said method being characterized in that a modulator of XRN1 is used. Also provided are uses of said method for therapeutical purposes, reagents therefore, as well as screening methods.

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Номер записи: 47

07-02-2013 дата публикации

Synthetic nanostructures including nucleic acids and/or other entities

Номер: US20130034599A1

Автор: Andrea Luthi, C. Shad Thaxton, Chad A. Mirkin, David M. Leander, Kaylin M. MCMAHON, Raja Kannan Mutharasan, Sushant Tripathy

Принадлежит: Northwestern University

Articles, compositions, kits, and methods relating to nanostructures, including synthetic nanostructures, are provided. Certain embodiments described herein include structures having a core-shell type arrangement; for instance, a nanostructure core may be surrounded by a shell including a material, such as a lipid bilayer, and may include other components such as oligonucleotides. In some embodiments, the structures, when introduced into a subject, can be used to deliver nucleic acids and/or can regulate gene expression. Accordingly, the structures described herein may be used to diagnose, prevent, treat or manage certain diseases or bodily conditions. In some cases, the structures are both a therapeutic agent and a diagnostic agent.

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Номер записи: 48

21-02-2013 дата публикации

Treatment of metastatic tumors

Номер: US20130045163A1

Автор: Abdellah Sentissi, Alison O'Neill, Douglas B. Jacoby, E. Michael Egan, Kamala Kesavan

Принадлежит: Morphotek Inc

The present invention is directed to methods and methods for the treatment, inhibition and/or reduction, and detection of metastatic tumors. In some embodiments, the inventive methods include systemic (e.g., intravenous) administration of a chlorotoxin agent that may or may not be labeled. In some embodiments, the inventive methods allow treatment, inhibition and/or reduction, and detection of metastases in the brain. In some embodiments, neovascularization is inhibited and/or newly formed vessels are caused to regress.

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Номер записи: 49

14-03-2013 дата публикации

Modulating production traits in avians

Номер: US20130067606A1

Автор: Andrew Henrik Sinclair, Craig Allen SMITH, John William Lowenthal, Robert John Moore, Timothy James Doran

Принадлежит: Australian Poultry CRC Pty Ltd

The present invention relates to methods of modulating traits, particularly production traits, in avians such as chickens. In particular, the invention relates to the in ovo delivery of a dsRNA molecule, especially siRNAs, to modify production traits in commercially important birds.

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Номер записи: 50

21-03-2013 дата публикации

Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure

Номер: US20130072671A1

Автор: Judith C. Van Deutekom

Принадлежит: Leids Universitair Medisch Centrum LUMC

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.

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Номер записи: 51

28-03-2013 дата публикации

Anti-HLA-DR Antibodies Suppress Allogeneic and Xenogeneic Immune Responses to Organ Transplants

Номер: US20130078263A1

Автор: Chien-Hsing Chang, David M. Goldenberg, Tokihiko SAWADA

Принадлежит: Immunomedics Inc

Disclosed herein are methods and compositions comprising anti-HLA-DR antibodies for treatment of allogeneic and xenogeneic immune responses occurring in organ transplant rejection and other immune dysfunction diseases. In preferred embodiments, the anti-HLA-DR antibodies are effective to deplete antigen-presenting cells, such as dendritic cells. Most preferably, administration of the therapeutic compositions depletes all subsets of APCs, including mDCs, pDCs, B cells and monocytes, without significant depletion of T cells. In alternative embodiments, administration of the therapeutic compositions suppresses proliferation of allo-reactive T cells, while preserving cytomegalovirus (CMV)-specific, CD8 + memory T cells. The compositions and methods provide a novel therapeutic agent for suppressing or preventing allogeneic or xenogeneic immune responses, without altering preexisting anti-viral immunity.

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Номер записи: 52

04-04-2013 дата публикации

Protein complementation regulators

Номер: US20130085095A1

Автор: Paul Terence Toran

Принадлежит: Boston University

The present invention relates to protein complementation regulators and methods for reducing target-independent interaction of protein complementation molecules.

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Номер записи: 53

02-05-2013 дата публикации

Method for the delivery of oligonucleotides

Номер: US20130108686A1

Автор: Francesc Xavier Blasco Sole, Jose Carlos Perales Losa, Ramon Eritja Casadella, Sandra Milena Ocampo

Принадлежит: Consejo Superior de Investigaciones Cientificas CSIC, Universitat de Barcelona UB

The invention relates to a method for obtaining formulations which improve the binding capacity of siRNAs in relation to plasma components, which promote the transport of siRNA in the blood, which increase the passage of siRNA through the cell membrane and, consequently, which increase the inhibitory activity of siRNA. The invention also relates to a method for obtaining a formulation comprising siRNA associated with plasma components, characterised in that the method includes a step in which the plasma components are dispersed in an aqueous medium.

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Номер записи: 54

02-05-2013 дата публикации

Microrna compositions and methods related thereto

Номер: US20130109628A1

Автор: Baicheng Hu, Walter J. Curran, Ya Wang

Принадлежит: EMORY UNIVERSITY

The disclosure relates to microRNAs (miRNAs) for the prophylaxis and/or treatment of neoplasia. The disclosure relates in particular to sequence corresponding to miR2 and the miR-548 family, including precursors, mature forms, fragments, and combinations thereof for the prophylaxis and/or treatment of neoplasias, particularly lung, stomach, and cervical cancer, alone or in combination with additional cancer treatments and therapeutics.

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Номер записи: 55

02-05-2013 дата публикации

Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna

Номер: US20130109740A1

Автор: Bob D. Brown, Henryk T. Dudek

Принадлежит: Dicerna Pharmaceuticals Inc

This invention relates to compounds, compositions, and methods useful for reducing β-catenin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

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Номер записи: 56

16-05-2013 дата публикации

Compositions and methods for silencing apolipoprotein b

Номер: US20130123339A1

Автор: Adam Judge, Alan Martin, Amy C.H. Lee, Ian Maclachlan, James Heyes, Marjorie Robbins, Mark Wood

Принадлежит: PROTIVA BIOTHERAPEUTICS INC

The present invention provides compositions and methods for the delivery of interfering RNAs such as siRNAs that silence APOB expression in cells such as liver cells. In particular, the nucleic acid-lipid particles provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells such as liver cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of APOB at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.

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Номер записи: 57

16-05-2013 дата публикации

Bioassay for gene silencing constructs

Номер: US20130125260A1

Автор: Ana M. Bailey, Charles Niblett

Принадлежит: Venganza Inc

The invention provides constructs and methods of screening for constructs useful in conferring resistance in plants to pests by gene silencing. The invention also provides pest-resistant plants transformed with the present constructs. One screening method of the invention comprises the steps of: selecting at least one pest target nucleotide sequence, producing a plurality of dsRNA test agents that target the pest target nucleotide sequence, testing and scoring the plurality of dsRNA test agents for toxicity to the pest, and producing a silencing construct based on a superior-scoring test agent.

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Номер записи: 58

23-05-2013 дата публикации

Targeted delivery to leukocytes using protein carriers

Номер: US20130129752A1

Автор: Dan Peer, Judy Lieberman, Motomu Shimaoka

Принадлежит: Immune Disease Institute Inc

Disclosed herein are is a leukocyte-selective delivery agent comprising, a targeting moiety that selectively binds LFA-I, a protein carrier moiety covalently linked to the targeting moiety, and a therapeutic agent associated with the carrier moiety. The delivery agent may be further selective for activated leukocytes, wherein the targeting moiety selectively binds LFA-I in its activated conformation. The targeting moiety comprises an antibody or functional fragment thereof, such as an scFV. Examples of antibodies or fragments thereof which selectively bind LFA-I activated conformation bind to the locked open I domain of LFA-I, or binds to the leg domain of the β2 subunit of LFA-I ((ILP2)—The antibody or functional fragment thereof may alternatively bind non-selectively to both low affinity and high affinity LFA-I. Examples of a non-protein carrier are a basic polypeptide such as protamine or a functional fragment thereof. One such fragment is RSQSRSRYYRQRQRSRRRRRRS. The therapeutic agent may comprise one or more of a nucleic acid, a small molecule, a polypeptide, and an antibody or functional fragment thereof. An example of a nucleic acid delivery agent comprises an RNA interference molecule. Examples of RNA interference molecules are siRNA, dsRNA, StRNA, shRNA, miRNA, and combinations thereof. Specific siRNAs are provided. Other examples of a nucleic acid delivery agent are a small RNA, an antagomir, an LNA, and an antisense oligonucleotide. Methods for leukocyte-selective delivery, or activated leukocyte-selective delivery in vivo, in vitro and ex vivo are also provided.

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Номер записи: 59

06-06-2013 дата публикации

Compositions for inhibiting gene expression and uses thereof

Номер: US20130142778A1

Автор: Ekambar Kandimalla, Ireneusz Nowak, Mallikarjuna Putta, Michael Reardon, Nicola LaMonica, Sudhir Agrawal, Tao Lan, Weiwen Jiang

Принадлежит: Idera Pharmaceuticals Inc

The inventors have examined the means for providing more efficacious miRNA blocking compounds. The inventors have discovered new structural features that surprisingly improve the efficacy of miRNA blocking molecules. These features include the presence of multiple 3′ ends and a linker at the 5′ ends. Surprisingly, these features improve the efficacy of the gene expression blocking compounds in a manner that decreases the compound's biologic instability. Even more surprisingly, this effect has been found to be applicable to both DNA and RNA oligonucleotide-based compounds and to have application in traditional antisense and RNAi technology.

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Номер записи: 60

06-06-2013 дата публикации

Therapeutic compositions

Номер: US20130144048A1

Автор: David Bumcrot, Kallanthottathil G. Rajeev, Muthiah Manoharan

Принадлежит: Alnylam Pharmaceuticals Inc

This application relates to therapeutic siRNA agents and methods of making and using the agents.

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Номер записи: 61

13-06-2013 дата публикации

BARD1 Isoforms in Lung and Colorectal Cancer and Use Thereof

Номер: US20130149711A1

Автор: Irmgard Irminger-Finger, yong-qiang Zhang

Принадлежит: HOPITAUX UNIVERSITAIRES DE GENEVE, UNIVERSITE DE GENEVE

The present invention relates to new BARD1 isoforms specific to lung cancer and colorectal cancer, a method for detecting thereof and a method for treating and/or preventing lung cancer and colorectal cancer.

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Номер записи: 62

20-06-2013 дата публикации

Lipid formulated single stranded rna

Номер: US20130156845A1

Автор: Eric E. Swayze, Kallanthottathil G. Rajeev, Muthiah Manoharan, Sayda Elbashir, Thazha P. Prakash, Walter F. Lima

Принадлежит: Alnylam Pharmaceuticals Inc, ISIS PHARMACEUTICALS INC

The present invention provides compositions comprising a nucleic acid lipid particle and an oligomeric compound and uses thereof. In certain embodiments, such compositions are useful as antisense compounds. Certain such antisense compounds are useful as RNase H antisense compounds or as RNAi compounds.

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Номер записи: 63

04-07-2013 дата публикации

Method for selective oligonucleotide modification

Номер: US20130172238A1

Автор: Andreas Mitsch, Anneliese Schneider, Bernd Betzler, Frank Jaschinski, Karl-Hermann Schlingensiepen

Принадлежит: Individual

Method for producing a modified oligonucleotide, wherein at least one polymer, preferably polyalkylene oxide, and/or a compound is covalently bound to the 5′-end or the 3′-end of the oligonucleotide via native ligation forming a native ligation site, with the proviso that the polymer and/or the compound is not a protein or peptide, if only the 5′-end of the oligonucleotide is modified by binding of the polymer or compound via native ligation. The invention is further directed to a modified oligonucleotide obtainable by the inventive method as well as the use of such modified oligonucleotide for the preparation of a medicament for preventing and/or treating a tumor, formation of metastasis, an immune disease or disorder, a cardiovascular disease or disorder, and/or a viral disease or disorder.

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Номер записи: 64

04-07-2013 дата публикации

Selective inhibition of polyglutamine protein expression

Номер: US20130172399A1

Автор: David R. Corey, Dinah W.Y. Sah, Jiaxin Hu

Принадлежит: Individual

The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntingtin Disease Protein and Ataxin-3 using double-stranded RNAs and nucleic acid analogs. Chemically-modified RNAs having at least one mismatch as compared to the target CAG repeat sequence are specifically contemplated.

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Номер записи: 65

18-07-2013 дата публикации

Ligand-conjugated monomers

Номер: US20130184328A1

Автор: Kallanthottathil G. Rajeev, Muthiah Manoharan, Venkitasamy Kesavan

Принадлежит: Alnylam Pharmaceuticals Inc

This invention relates composition and methods for making and using chemically modified oligonucleotides agents for inhibiting gene expression.

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Номер записи: 66

01-08-2013 дата публикации

Methods and compositions for improving sleep and memory

Номер: US20130195763A1

Автор: Charles F. Landry, Jason R. Gerstner, Jerry C. Yin

Принадлежит: Individual

A method for determining whether a substance can increase the expression level of a fatty acid binding protein (FABP) in an animal. The method includes using a cell that includes an expression construct that comprises a FABP promoter operably linked to a polynucleotide sequence encoding a reporter molecule, wherein the cell is contacted with a candidate substance and then cultivating the cell under conditions conducive to expression of the reporter molecule. Increased expression of the construct in the presence of the candidate substance as compared to a control leads to improved sleep and long-term memory in the subject.

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Номер записи: 67

01-08-2013 дата публикации

ENHANCEMENT OF siRNA SILENCING ACTIVITY USING UNIVERSAL BASES OR MISMATCHES IN THE SENSE STRAND

Номер: US20130196434A1

Автор: Haripriya Addepalli, Martin Maier, Muthiah Manoharan

Принадлежит: Alnylam Pharmaceuticals Inc

One aspect of the present invention relates to a double stranded nucleic acid useful as an siRNA, that has a sense strand and an antisense strand relative to a target nucleic acid, where the sense strand contains one or more modified nucleobases, or one or more mismatch base pairings with the antisense strand. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one nucleoside comprising a non-natural nucleobase. Another aspect of the invention relates to a method of gene silencing, comprising administering to a mammal in need thereof a therapeutically effective amount of a double-stranded oligonucleotides containing a sense strand and an antisense strand, where the sense strand contains one or more modified nucleobases, or one or more mismatch base pairings with the antisense strand.

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Номер записи: 68

08-08-2013 дата публикации

Asymmetric biofunctional silyl monomers and particles thereof as prodrugs and delivery vehicles for pharmaceutical, chemical and biological agents

Номер: US20130203675A1

Автор: Ashish Pandya, Joseph M. Desimone, Mary Napier, Matthew Finniss, Matthew Parrott

Принадлежит: Individual

Asymmetric bifunctional silyl (ABS) monomers comprising covalently linked pharmaceutical, chemical and biological agents are described. These agents can also be covalently bound via the silyl group to delivery vehicles for delivering the agents to desired targets or areas. Also described are delivery vehicles which contain ABS monomers comprising covalently linked agents and to vehicles that are covalently linked to the ABS monomers. The silyl modifications described herein can modify properties of the agents and vehicles, thereby providing desired solubility, stability, hydrophobicity and targeting.

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Номер записи: 69

08-08-2013 дата публикации

PREPARATION OF MICROVESICLE-siRNA COMPLEXES AND USE THEREOF IN AIDS TREATMENT

Номер: US20130203837A1

Автор: Chenyu Zhang, Hongwei Gu, Ke Zeng, Liming Li, Minghui Cao

Принадлежит: Micromedmark Biotech Co Ltd

The present invention provides drugs for treating AIDS, which comprises microvesicles carrying anti-HIV specific siRNA. The present invention also provides a preparation method of the drug.

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Номер записи: 70

15-08-2013 дата публикации

siRNA Targeting Myeloid Differentiation Primary Response Gene (88) (MYD88)

Номер: US20130210676A1

Автор: Anastasia Khvorova, Angela Reynolds, Devin Leake, Stephen Scaringe, Steven Read, William Marshall

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 MYD88.

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Номер записи: 71

22-08-2013 дата публикации

Preventive or therapeutic agent for fibrosis

Номер: US20130217754A1

Автор: Ayako Fukuda, Esteban C. Gabazza, Hidekazu Toyobuku, Tetsu Kobayashi, Tetsuya Hasegawa

Принадлежит: Mie University NUC, Otsuka Pharmaceutical Co Ltd

Provided is siRNA effective for the treatment of fibrosis and a pharmaceutical containing the siRNA. An siRNA having a full length of 30 or fewer nucleotides and targeting a sequence consisting of 17 to 23 consecutive bases selected from the group consisting of bases at positions 1285 to 1318, bases at positions 1398 to 1418, bases at positions 1434 to 1463, bases at positions 1548 to 1579, bases at positions 1608 to 1628, bases at positions 1700 to 1726, bases at positions 1778 to 1798, bases at positions 1806 to 1826, and bases at positions 1887 to 1907 of SEQ ID NO: 1.

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Номер записи: 72

29-08-2013 дата публикации

siRNA Compositions and Methods for Potently Inhibiting Viral Infection

Номер: US20130225651A1

Автор: Bojiian Zheng, Hongyan Sui, Yongping Lin

Принадлежит: University of Hong Kong HKU

No antiviral regimen has been consistently successful in treating H5N1 virus infection. We demonstrate that a group of highly effective siRNAs targeting different H5N1 viral genes shares a unique motif, GGAGU/ACUCC. We further demonstrate that the effectiveness of siRNAs containing this motif is not sequence specific. The results suggested that the structure of the unique motif is critical in determining the potency of siRNA-mediated protective effects against viral infection and this potent in vivo protection is associated with early productions of β-defensin and IL-6 induced by the motif. Provided are methods and prophylactic and therapeutic agents useful against other viral infections in addition to the H5N1 influenza virus.

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Номер записи: 73

26-09-2013 дата публикации

Protease degradable polypeptides and uses thereof

Номер: US20130251640A1

Автор: Ching-Hsuan Tung, Wael R. Abd-Elgaliel

Принадлежит: Methodist Hospital System

Provided herein are polypeptides that are selectively cleaved by cathepsin E. Also provided are methods of detecting cathepsin E. The methods comprise contacting cathepsin E with the polypeptides provided herein and detecting fluorescence. Further provided are methods of diagnosing cancer or pre-cancerous conditions in a subject. Also provided herein is a multilayered nanoparticle or a composition comprising the multilayer nanoparticle, wherein the multilayered nanoparticle comprises a negatively charged nanoparticle core or capsule coated with alternating positive and negative layers. Optionally, the positive layer comprises a positively charged protease degradable polypeptide. Optionally, the negative layer comprises a negatively charged therapeutic agent or a therapeutic agent and a means for providing the agent with a negative charge. For example, optionally, the therapeutic agent is linked to a negatively charged polymer. Further provided are methods of treating or preventing a disease characterized by expression of a protease in a subject using the nanoparticle.

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Номер записи: 74

26-09-2013 дата публикации

Methods of determining potency of chemically-synthesized oligonucleotides

Номер: US20130252243A1

Автор: Stanley T. Crooke, Timothy Vickers, Walter F. Lima

Принадлежит: ISIS PHARMACEUTICALS INC

Provided herein are methods for determining potency of RNAi agents. Such methods include, but are not limited to, cell-based and cell-free assays that measure binding of an RNAi agent with Ago2 or that measure Ago2 activity in the presence of such RNAi agents. Also provided are assays that determine potency of RNAi agents by assessing their ability to compete with other RNAi agents, including control RNAi agents, for binding and/or activation of Ago2.

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Номер записи: 75

26-09-2013 дата публикации

Novel single chemical entities and methods for delivery of oligonucleotides

Номер: US20130253168A1

Автор: Anthony W. Shaw, Daniel Zewge, David M. Tellers, Francis Gosselin, Steven L. Colletti, Thomas J. Tucker, Vasant R. Jadhav, Yu Yuan

Принадлежит: Individual

In an embodiment the instant invention discloses a modular composition comprising 1) an oligonueleotide; 2) one or more linkers, which may be the same or different, selected from Table 1, wherein the linkers are attached to the oligonucleotide at the 2′-position of the ribose rings and/or the terminal 3′- and/or 5′-positions of the oligonucleotide; 3) optionally, one or more peptides, which may be the same or different, selected from SEQ ID NOs: 1-59, wherein the peptides are attached to the linkers; and optionally one or more lipids, solubilizing groups and/or targeting ligands attached to the oligonucleotide.

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Номер записи: 76

26-09-2013 дата публикации

Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Номер: US20130253180A1

Автор: Graham McClorey, Stephen Donald Wilton, Sue Fletcher

Принадлежит: University of Western Australia

Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.

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Номер записи: 77

10-10-2013 дата публикации

Gene Silencing by Single-Stranded Polynucleotides

Номер: US20130267577A1

Автор: Gretchen M. Unger

Принадлежит: GeneSegues Inc

The present invention relates to compositions and methods for concurrently activating antisense and double-stranded RNase (dsRNase) mechanisms for inhibiting expression of a targeted gene, by delivering a single stranded bifunctional chimeric DNA/RNA oligonucleotide optimized for siRNA activity as well as antisense activity, into the nucleus of a target cell.

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Номер записи: 78

10-10-2013 дата публикации

Immune regulatory oligonucleotide (iro) compounds to modulate toll-like receptor based immune response

Номер: US20130267583A1

Автор: Daqing Wang, Dong Yu, Ekambar R. Kandimalla, FuGang Zhu, Lakshmi Bhagat, Sudhir Agrawal, Yukui Li

Принадлежит: Idera Pharmaceuticals Inc

The invention provides novel immune regulatory oligonucleotides (IRO) as antagonist of TLRs and methods of use thereof. These IROs have unique sequences that inhibit or suppress TLR-mediated signaling in response to a TLR ligand or TLR agonist. The methods may have use in the prevention and treatment of cancer, an autoimmune disorder, airway inflammation, inflammatory disorders, infectious disease, skin disorders, allergy, asthma or a disease caused by a pathogen.

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Номер записи: 79

17-10-2013 дата публикации

Tyrosine-phosphorylated wbp2, a novel cancer target and biomarker

Номер: US20130273056A1

Автор: Yoon Pin Lim

Принадлежит: NATIONAL UNIVERSITY OF SINGAPORE

WW-binding protein 2 (WBP2) has been demonstrated in different studies to be a tyrosine kinase substrate, to activate ERα/PR transcription and to play a role in breast cancer. However, the role of WBP2 tyrosine phosphorylation in regulating ER function and breast cancer biology is unknown. Here, we established WBP2 as a tyrosine phosphorylation target of estrogen signaling via EGFR crosstalk. Using dominant negative, constitutively active mutants, RNAi and pharmacological studies, we demonstrated that phosphorylation of WBP2 at Tyr192 and Tyr231 could be regulated by c-Src and c-Yes kinases. We further showed that abrogating WBP2 phosphorylation impaired >60% of ERα reporter activity putatively by blocking nuclear entry of WBP2 and its interaction with ERα. Compared to vector control, overexpression of WBP2 and its phospho-mimic mutant in MCF7 resulted in larger tumors in mice, induced loss of cell-cell adhesion, enhanced cell proliferation, anchorage-independent growth, migration and invasion in both estrogen-dependent and-independent manner, events of which could be substantially abolished by overexpression of phosphorylation-defective mutant. Wnt/β-catenin inhibitor FH535 blocked phospho-WBP2-mediated cancer cell growth more pronouncedly than tamoxifen and fulvestrant, in part by reducing the expression of ERα.

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Номер записи: 80

17-10-2013 дата публикации

Analytical device and analytical method

Номер: US20130273530A1

Автор: Iwao Waga, Jou Akitomi, Katsunori Horii, Shintarou Katou

Принадлежит: NEC Solution Innovators Ltd

The present invention provides a technique capable of simply analyzing a target to be analyzed. An analytical device of the present invention includes a basal plate; a nucleic acid element; and a detection section of detecting a signal. The nucleic acid element and the detection section are arranged on the basal plate. The nucleic acid element includes a first nucleic acid molecule and a second nucleic acid molecule. The first nucleic acid molecule is a nucleic acid molecule capable of binding to a target. The second nucleic acid molecule is a nucleic acid molecule capable of binding to streptavidin. When the target does not bind to the first nucleic acid molecule, a binding capacity of the second nucleic acid molecule to the streptavidin is inactivated. When the target binds to the first nucleic acid molecule, a binding capacity of the second nucleic acid molecule to the streptavidin is activated. The detection section detects binding between the second nucleic acid molecule and the streptavidin. The target is bound to the first nucleic acid molecule, so that the streptavidin is bound to the second nucleic acid molecule. Thus, the target can be analyzed through detecting the binding between the second nucleic acid molecule and the streptavidin using the detection device.

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Номер записи: 81

17-10-2013 дата публикации

Low molecular weight cationic lipids for oligonucleotide delivery

Номер: US20130274523A1

Автор: John A. Bawiec, Iii, Zhengwu J. Deng

Принадлежит: Individual

The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.

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Номер записи: 82

24-10-2013 дата публикации

Compositions and methods for inhibiting expression of the alas1 gene

Номер: US20130281511A1

Автор: Brian Bettencourt, Kevin Fitzgerald, Makiko Yasuda, Robert J. Desnick, William Querbes

Принадлежит: Individual

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

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Номер записи: 83

14-11-2013 дата публикации

Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger rna

Номер: US20130302295A1

Автор: Jiwu Wang, Luigi Warren, Yuhui NI

Принадлежит: Allele Biotechnology and Pharmaceuticals, Inc.

The present disclosure relates generally to novel methods and compositions for using engineered reprogramming factor(s) for the creation of induced pluripotent stem cells (iPSCs) through a kinetically controlled process. Specifically, this disclosure relates to establishing combinations of reprogramming factors, including fusions between conventional reprogramming factors with transactivation domains, optimized for reprogramming various types of cells. More specifically, the exemplary methods disclosed herein can be used for creating induced pluripotent stem cells from various mammalian cell types, including human fibroblasts. Exemplary methods of feeder-free derivation of human induced pluripotent stem cells using synthetic messenger RNA are also disclosed.

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Номер записи: 84

14-11-2013 дата публикации

Design of rnai molecules with non-watson crick pairing based on artificial mutation consensus sequences to counter escape mutations

Номер: US20130303589A1

Автор: John J. Rossi, Ulrike Jung

Принадлежит: CITY OF HOPE

Universal RNA interference (RNAi) molecules having an inhibitory RNA sequence which binds a target pathologic RNA sequence are provided according to some embodiments. Such RNAi molecules bind the target pathologic RNA sequence via at least one non-Watson Crick paired base. In some embodiments, the target pathologic RNA sequence is a target viral RNA sequence derived from a human immunodeficiency HIV virus, a hepatitis B virus (HBV), a hepatitis C virus (HCV), or an influenza virus.

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Номер записи: 85

28-11-2013 дата публикации

Methods and pharmaceutical compositions for the treatment of heart failure

Номер: US20130315932A1

Автор: Frédéric JAISSER

Принадлежит: Institut National de la Sante et de la Recherche Medicale INSERM

The present invention relates to an inhibitor of NGAL gene expression or a NGAL antagonist for use in the prevention or the treatment of heart failure.

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Номер записи: 86

28-11-2013 дата публикации

Nucleic acids encoding peptides for treating wounds, anti-angiogenic compounds and uses thereof

Номер: US20130316956A1

Автор: Ira M. Herman

Принадлежит: TUFTS UNIVERSITY

The present disclosure relates to protein and peptide chemistry. More particularly, it relates to compounds, compositions and uses thereof for promoting and inhibiting angiogenesis. The peptides of the present disclosure include peptides comprising SEQ ID NOs: 1-4 which promote angiogenesis and cell proliferation. Further, the anti-angiogenic compounds of the present disclosure include antisense oligonucleotides that hybridize or are complementary to the polynucleotides of SEQ ID NOs: 5-16, and the like.

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Номер записи: 87

12-12-2013 дата публикации

Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Номер: US20130331438A1

Автор: Graham McClorey, Stephen Donald Wilton, Sue Fletcher

Принадлежит: University of Western Australia

An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.

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Номер записи: 88

12-12-2013 дата публикации

Methods for Diagnosing Stomach Cancer Using MicroRNAs

Номер: US20130331439A1

Автор: Carlo M. Croce, George A. Calin, Stefano Volinia

Принадлежит: Ohio State University

Described herein are methods for diagnosing stomach cancer using microRNAs. Also described are methods and compositions for the diagnosis and treatment of solid cancers. Methods of identifying inhibitors of tumorigenesis are also provided.

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Номер записи: 89

26-12-2013 дата публикации

Human antibodies to the glucagon receptor

Номер: US20130344538A1

Автор: Haruka Okamoto, Joyce Harp, Mark Sleeman

Принадлежит: Regeneron Pharmaceuticals Inc

The present invention provides antibodies that bind to the human glucagon receptor, designated GCGR and methods of using same. According to certain embodiments of the invention, the antibodies are fully human antibodies that bind to human GCGR. The antibodies of the invention are useful for lowering blood glucose levels and blood ketone levels and are also useful for the treatment of diseases and disorders associated with one or more GCGR biological activities, including the treatment of diabetes, diabetic ketoacidosis and long-term complications associated with diabetes, or other metabolic disorders characterized in part by elevated blood glucose levels.

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Номер записи: 90

02-01-2014 дата публикации

Chimeric oligomeric compounds for modulation of splicing

Номер: US20140005374A1

Автор: C. Frank Bennett, Casey C. Kopczynski, Nicholas M. Dean, Ryszard M. Kole

Принадлежит: ISIS PHARMACEUTICALS INC, Sarepta Therapeutics Inc

Disclosed herein are compounds, compositions and methods for modulating splicing of a selected target mRNA. Further provided are uses of the disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Methods of enhancing cellular uptake, modulating tissue distribution and enhancing pharmacological activity of RNase H-independent antisense oligonucleotides are also provided.

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Номер записи: 91

16-01-2014 дата публикации

MicroRNAs and uses thereof

Номер: US20140017780A1

Автор: Amir Avniel, Itzhak Bentwich, Ranit Aharonov, Yael Karov

Принадлежит: Rosetta Genomics Ltd

Described herein are novel polynucleotides associated with prostate and lung cancer. The polynucleotides are miRNAs and miRNA precursors. Related methods and compositions that can be used for diagnosis, prognosis, and treatment of those medical conditions are disclosed. Also described herein are methods that can be used to identify modulators of prostate and lung cancer.

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Номер записи: 92

16-01-2014 дата публикации

LOW DENSITY LIPOPROTEIN RECEPTOR-MEDIATED siRNA DELIVERY

Номер: US20140018296A1

Автор: Jon E. Chatterton

Принадлежит: Alcon Research LLC

The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo. The conjugates comprise a ligand that can bind to a low density lipoprotein receptor (LDLR) or LDLR family member. Therapeutic uses for the conjugates are also provided.

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Номер записи: 93

16-01-2014 дата публикации

Methods for Diagnosing Prostate Cancer using MicroRNAs

Номер: US20140018411A1

Автор: Carlo M. Croce, George A. Calin, Stefano Volinia

Принадлежит: Ohio State University

Described herein are methods for diagnosing prostate cancer using microRNAs. Also described are methods and compositions for the diagnosis and treatment of solid cancers. Methods of identifying inhibitors of tumorigenesis are also provided.

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Номер записи: 94

23-01-2014 дата публикации

Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna

Номер: US20140024698A1

Автор: Francis S. Collins, Kan CAO, Michael R. Erdos, Ryszard Kole

Принадлежит: Sarepta Therapeutics Inc, University of Maryland at Baltimore, US Department of Health and Human Services

Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

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Номер записи: 95

30-01-2014 дата публикации

Methods and compositions involving mirna and mirna inhibitor molecules

Номер: US20140031415A1

Автор: Angie Cheng, David Brown, Dmitriy Ovcharenko, Eric Devroe, Kevin Kelnar, Lance Ford, Mike Byrom, Rich Jarvis

Принадлежит: Asuragen Inc

The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.

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Номер записи: 96

13-02-2014 дата публикации

Methods and compositions for the diagnosis, classification, and treatment of cancer

Номер: US20140045920A1

Автор: Ajay Pratap Singh, Sanjeev Srivastava, Seema Singh

Принадлежит: UNIVERSITY OF SOUTH ALABAMA

Some embodiments of the present technology relate to methods and compositions for the diagnosis and treatment of cancer. Some embodiments include methods and compositions for the diagnosis and treatment of castration-resistant prostate cancer. Some embodiments include methods and compositions for the diagnosis and treatment of pancreatic cancer.

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Номер записи: 97

20-03-2014 дата публикации

Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Номер: US20140080898A1

Автор: Graham McClorey, Stephen Donald Wilton, Sue Fletcher

Принадлежит: University of Western Australia

Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.

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Номер записи: 98

27-03-2014 дата публикации

Compounds and methods for altering activin receptor-like kinase signaling

Номер: US20140088174A1

Автор: Adriana Marie Rus, Dwi Utami KEMALADEWI, Peter Abraham Christiaan "T Hoen, Peter Ten Duke, Wilhelmus Martinus Hendrikus HOOGAARS

Принадлежит: Leids Universitair Medisch Centrum LUMC

Described are compounds and methods useful in the promotion of muscle growth, the treatment of muscle loss or insufficient muscle growth, and the treatment of fibrotic conditions.

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Номер записи: 99

07-01-2016 дата публикации

METHODS FOR CLASSIFYING A CANCER AS SUSCEPTIBLE TO TMEPAI-DIRECTED THERAPIES AND TREATING SUCH CANCERS

Номер: US20160000735A1

Автор: SAIKUMAR Pothana, SINGHA Prajjal Kanti

Принадлежит:

The invention provides methods for classifying a cancer as susceptible to transmembrane prostate androgen induced (TMEPAI)-directed therapies, and methods for treating such cancers. The field of the invention pertains generally to medicine, pathology and oncology. More particularly, it addresses the treatment of breast cancer, such as triple-negative breast cancer, using a transmembrane prostate androgen induced (TMEPAI)-directed therapy. 1. A method of inhibiting a cancer cell that overexpresses transmembrane prostate androgen induced (TMEPAI) compared to normal control cell comprising contacting the cancer cell with an agent that inhibits TMEPAI expression or activity.2. The method of claim 1 , wherein the cancer cell is selected from a brain cancer cell claim 1 , a prostate cancer cell claim 1 , a lung cancer cell claim 1 , a head & neck cancer cell claim 1 , a esophageal cancer cell claim 1 , a pancreatic cancer cell claim 1 , a stomach cancer cell claim 1 , a liver cancer cell claim 1 , a colon cancer cell claim 1 , a ovarian cancer cell claim 1 , a cervical cancer cell claim 1 , a testicular cancer cell claim 1 , or a skin cancer cell.3. The method of claim 2 , wherein the cancer cell is a breast cancer cell.4. The method of claim 3 , wherein the breast cancer cell is a triple-negative breast cancer cell.5. The method of claim 1 , wherein the agent is an siRNA or an antisense molecule.6. The method of claim 1 , wherein the agent is an intrabody.7. The method of claim 1 , wherein the agent is a small molecule.8. The method of claim 7 , wherein the agent is a triterpenoid.9. The method of claim 1 , further comprising contacting said cell with a second anti-cancer agent or therapy.10. The method of claim 1 , wherein said cancer cell is metastatic claim 1 , recurrent or multi-drug resistant.11. A method of treating a subject with cancer claim 1 , wherein cells of said cancer overexpress transmembrane prostate androgen induced (TMEPAI) compared to normal cells ...

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Номер записи: 100

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