KR20140084232A - Gccr 발현의 안티센스 조절 - Google Patents

Gccr 발현의 안티센스 조절 Download PDF

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Publication number
KR20140084232A
KR20140084232A KR1020147013594A KR20147013594A KR20140084232A KR 20140084232 A KR20140084232 A KR 20140084232A KR 1020147013594 A KR1020147013594 A KR 1020147013594A KR 20147013594 A KR20147013594 A KR 20147013594A KR 20140084232 A KR20140084232 A KR 20140084232A
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compound
modified
seq
gccr
linked nucleosides
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Korean (ko)
Inventor
수잔 엠. 프라이어
Sanjay Bhanot
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아이시스 파마수티컬즈 인코포레이티드
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
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    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Hematology (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020147013594A 2011-10-25 2012-10-25 Gccr 발현의 안티센스 조절 Withdrawn KR20140084232A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161551378P 2011-10-25 2011-10-25
US61/551,378 2011-10-25
PCT/US2012/061984 WO2013063313A1 (en) 2011-10-25 2012-10-25 Antisense modulation of gccr expression

Publications (1)

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KR20140084232A true KR20140084232A (ko) 2014-07-04

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KR1020147013594A Withdrawn KR20140084232A (ko) 2011-10-25 2012-10-25 Gccr 발현의 안티센스 조절

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US (2) US8901098B2 (enExample)
EP (1) EP2771463A4 (enExample)
JP (1) JP2015501155A (enExample)
KR (1) KR20140084232A (enExample)
CN (1) CN103906838A (enExample)
AU (2) AU2012328680A1 (enExample)
BR (1) BR112014009790A2 (enExample)
CA (1) CA2853373A1 (enExample)
HK (1) HK1201555A1 (enExample)
IL (1) IL232183A0 (enExample)
IN (1) IN2014CN03749A (enExample)
MX (1) MX350944B (enExample)
RU (1) RU2014119787A (enExample)
WO (1) WO2013063313A1 (enExample)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102482890B1 (ko) 2013-05-01 2022-12-30 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법
CN110903337A (zh) 2014-05-01 2020-03-24 Ionis制药公司 用于调节生长激素受体表达的组合物和方法
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
JP6606557B2 (ja) * 2015-04-16 2019-11-13 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｃ９ｏｒｆ７２発現を調節するための組成物
CN108026529A (zh) * 2015-09-30 2018-05-11 Ionis制药公司 组合疗法
US10083668B2 (en) 2016-03-09 2018-09-25 Semiconductor Energy Laboratory Co., Ltd. Semiconductor device, display device, and electronic device
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
SG11202003488WA (en) 2017-10-20 2020-05-28 Dicerna Pharmaceuticals Inc Methods for treating hepatitis b infection
KR20200109311A (ko) 2018-01-16 2020-09-22 다이서나 파마수이티컬, 인크. Aldh2 발현을 억제하기 위한 조성물 및 방법
MX2021009754A (es) 2019-02-12 2021-09-08 Dicerna Pharmaceuticals Inc Metodos y composiciones para inhibir la expresion de cyp27a1.
CN109680095A (zh) * 2019-02-25 2019-04-26 广西中医药大学 一种鉴定苦玄参品种的ssr引物组合物及其品种鉴定方法
CA3135402A1 (en) 2019-04-04 2020-10-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system
MX2022011550A (es) 2020-03-18 2023-01-04 Dicerna Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión de angptl3.
AU2021321430A1 (en) 2020-08-04 2023-03-02 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting PLP1 expression
MX2023001541A (es) 2020-08-04 2023-03-08 Dicerna Pharmaceuticals Inc Administracion sistemica de oligonucleotidos.
TW202221120A (zh) 2020-08-04 2022-06-01 美商黛瑟納製藥公司 用於治療代謝症候群之組成物及方法
IL300338A (en) 2020-08-05 2023-04-01 Dicerna Pharmaceuticals Inc Compounds and methods for inhibiting the expression of LPA
US20240294907A1 (en) 2020-10-08 2024-09-05 Dicerna Pharmaceuticals, Inc. Selective delivery of oligonucleotides to glial cells
CA3214439A1 (en) 2021-04-12 2022-10-20 Boehringer Ingelheim International Gmbh Compositions and methods for inhibiting ketohexokinase (khk)
WO2022221430A1 (en) 2021-04-14 2022-10-20 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating pnpla3 expression
WO2022223515A2 (en) 2021-04-19 2022-10-27 Novo Nordisk A/S Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
AR125992A1 (es) 2021-05-28 2023-08-30 Novo Nordisk As Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1)
US20230107967A1 (en) 2021-08-25 2023-04-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting alpha-1 antitrypsin expression
TW202330920A (zh) 2021-12-01 2023-08-01 美商戴瑟納製藥股份有限公司 用於調節ａｐｏｃ３表現之組合物及方法
US20230374522A1 (en) 2022-04-15 2023-11-23 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating scap activity
AU2023269281A1 (en) 2022-05-12 2024-10-10 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting mapt expression
IL316843A (en) 2022-05-13 2025-01-01 Dicerna Pharmaceuticals Inc Compounds and methods for inhibiting SNCA deactivation
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
TW202430637A (zh) 2022-11-16 2024-08-01 美商戴瑟納製藥股份有限公司 Stat3靶向性寡核苷酸及其用途
WO2025029625A1 (en) 2023-07-28 2025-02-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for programmed death ligand receptor (pd-l1) expression

Family Cites Families (98)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4426330A (en) 1981-07-20 1984-01-17 Lipid Specialties, Inc. Synthetic phospholipid compounds
US4534899A (en) 1981-07-20 1985-08-13 Lipid Specialties, Inc. Synthetic phospholipid compounds
WO1988000975A1 (en) 1986-08-01 1988-02-11 Genetics Institute, Inc. High level inducible expression of heterologous genes
US5354844A (en) 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5227170A (en) 1989-06-22 1993-07-13 Vestar, Inc. Encapsulation process
US5527528A (en) 1989-10-20 1996-06-18 Sequus Pharmaceuticals, Inc. Solid-tumor treatment method
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5356633A (en) 1989-10-20 1994-10-18 Liposome Technology, Inc. Method of treatment of inflamed tissues
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
US5469854A (en) 1989-12-22 1995-11-28 Imarx Pharmaceutical Corp. Methods of preparing gas-filled liposomes
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
JP3220180B2 (ja) 1991-05-23 2001-10-22 三菱化学株式会社 薬剤含有タンパク質結合リポソーム
NZ244306A (en) 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5521291A (en) 1991-09-30 1996-05-28 Boehringer Ingelheim International, Gmbh Conjugates for introducing nucleic acid into higher eucaryotic cells
US5872242A (en) 1992-10-05 1999-02-16 Isis Pharmaceuticals, Inc. Antisense oligonucleotide inhibition of ras
US5985558A (en) 1997-04-14 1999-11-16 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos
US5583020A (en) 1992-11-24 1996-12-10 Ribozyme Pharmaceuticals, Inc. Permeability enhancers for negatively charged polynucleotides
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
JP3351476B2 (ja) 1993-01-22 2002-11-25 三菱化学株式会社 リン脂質誘導体及びそれを含有するリポソーム
US5395619A (en) 1993-03-03 1995-03-07 Liposome Technology, Inc. Lipid-polymer conjugates and liposomes
US5462854A (en) 1993-04-19 1995-10-31 Beckman Instruments, Inc. Inverse linkage oligonucleotides for chemical and enzymatic processes
US5534259A (en) 1993-07-08 1996-07-09 Liposome Technology, Inc. Polymer compound and coated particle composition
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5417978A (en) 1993-07-29 1995-05-23 Board Of Regents, The University Of Texas System Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5591721A (en) 1994-10-25 1997-01-07 Hybridon, Inc. Method of down-regulating gene expression
US5512295A (en) 1994-11-10 1996-04-30 The Board Of Trustees Of The Leland Stanford Junior University Synthetic liposomes for enhanced uptake and delivery
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US5877309A (en) 1997-08-13 1999-03-02 Isis Pharmaceuticals, Inc. Antisense oligonucleotides against JNK
US6133246A (en) 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
KR100414936B1 (ko) 1997-09-12 2004-01-13 엑시콘 에이/에스 이환 및 삼환 뉴클레오시드, 뉴클레오타이드 및올리고뉴클레오타이드 동족체
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6248724B1 (en) 1997-09-25 2001-06-19 University Of Florida Antisense oligonucleotide compositions targeted to angiotensin converting enzyme MRNA and methods of use
US20030203862A1 (en) 1998-03-26 2003-10-30 Miraglia Loren J. Antisense modulation of MDM2 expression
US6238921B1 (en) 1998-03-26 2001-05-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of human mdm2 expression
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6821724B1 (en) 1998-09-17 2004-11-23 Affymetrix, Inc. Methods of genetic analysis using nucleic acid arrays
US6228642B1 (en) 1998-10-05 2001-05-08 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of tumor necrosis factor-(α) (TNF-α) expression
IL144975A0 (en) 1999-02-26 2002-06-30 Univ British Columbia A composition containing an antisense oligonucleotide
US6900187B2 (en) 1999-02-26 2005-05-31 The University Of British Columbia TRPM-2 antisense therapy using an oligonucleotide having 2′-O-(2-methoxy)ethyl modifications
GB9907048D0 (en) 1999-03-27 1999-05-19 Karobio Ab Novel glucocorticoid receptor ligands for the treatment of meta bolic disorders
PT1178999E (pt) 1999-05-04 2007-06-26 Santaris Pharma As Análogos de l-ribo-lna
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
EP1237925B1 (en) 1999-12-07 2007-07-11 Sumitomo Chemical Company, Limited Mutant er-alpha and test systems for transactivation
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
GB0008936D0 (en) 2000-04-11 2000-05-31 Glaxo Group Ltd Vectors
US6649341B1 (en) 2000-04-19 2003-11-18 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Human glucocorticoid receptor 1A promoter and splice variants
US6656700B2 (en) 2000-05-26 2003-12-02 Amersham Plc Isoforms of human pregnancy-associated protein-E
US7227007B2 (en) 2000-12-28 2007-06-05 Asahi Kasei Pharma Corporation NF-κB activating gene
WO2003008583A2 (en) 2001-03-02 2003-01-30 Sagres Discovery Novel compositions and methods for cancer
WO2002096943A1 (en) 2001-05-25 2002-12-05 Asahi Kasei Kabushiki Kaisha Stat6-activating genes
US20030092616A1 (en) 2001-05-25 2003-05-15 Akio Matsuda STAT6 activation gene
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
WO2003008578A2 (en) 2001-07-20 2003-01-30 Board Of Trustees Of The University Of Illinois Reagents and methods for identifying gene targets for treating cancer
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US20060088828A1 (en) 2002-01-23 2006-04-27 Harris Peter C Polycystic kidney disease nucleic acids and proteins
AU2003213054A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF POLYCOMB GROUP PROTEIN EZH2 GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
AU2003213057A1 (en) 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Incoporated Rna interference mediated inhibition of checkpoint kinase-1 (chk-1) gene expression using short interfering nucleic acid
JP2003265184A (ja) 2002-03-18 2003-09-24 Kokuritsu Iyakuhin Shokuhin Eisei Kenkyusho 被検者におけるグルココルチコイド製剤の有効性の検査方法
BR0308923A (pt) 2002-03-27 2005-01-04 Aegera Therapeutics Inc OligÈmeros de nucleobase de iap anti-sentido e empregos destes
ES2649817T3 (es) 2002-04-05 2018-01-15 Roche Innovation Center Copenhagen A/S Compuestos oligom¿¿ricos para la modulaci¿®n de la expresi¿®n de HIF-1¿Á
AU2003233634A1 (en) 2002-05-20 2003-12-12 Pharmacia Corporation Antisense modulation of glucocorticoid receptor expression
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
KR101076146B1 (ko) 2002-08-16 2011-10-21 렉산 팔마큐티칼스, 인크. 사람의 Ａｋｔ-1 유전자의 발현을 저해하는 안티센스 올리고뉴클레오타이드의 용도
WO2004034969A2 (en) 2002-08-19 2004-04-29 Regulome Corporation Functional sites
EP1530636B1 (en) 2002-08-21 2010-08-18 The University Of British Columbia Treatment of melanoma by reduction in clusterin levels
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1560840B1 (en) 2002-11-05 2015-05-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
AU2003281988B2 (en) 2002-11-25 2008-06-12 University Of Copenhagen Porcine polymorphisms and methods for detecting them
AU2003224132A1 (en) 2003-04-24 2004-11-19 Galapagos Genomics N.V. Effective sirna knock-down constructs
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
JP4731324B2 (ja) 2003-08-28 2011-07-20 武 今西 Ｎ−ｏ結合性架橋構造型新規人工核酸
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US8012944B2 (en) 2003-10-30 2011-09-06 Pharmascience Inc. Method for treating cancer using IAP antisense oligomer and chemotherapeutic agent
CN100569945C (zh) 2003-12-23 2009-12-16 桑塔里斯制药公司 用于调节bcl-2的寡聚化合物
JP2007520222A (ja) 2004-01-20 2007-07-26 アイシス ファーマシューティカルズ インコーポレイテッド グルココルチコイドレセプター発現の調節
EP2397563A3 (en) 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
US20080125583A1 (en) 2005-02-11 2008-05-29 International Business Machines Corporation Ribonucleic acid interference molecules
JP2006320271A (ja) * 2005-05-20 2006-11-30 Tokyo Institute Of Technology 低分子化合物とタンパク質の結合評価方法
CA2623772A1 (en) * 2005-09-19 2007-03-29 Johnson & Johnson Pharmaceutical Research & Development, L.L.C. Modulation of glucocorticoid receptor expression
ES2516815T3 (es) 2006-01-27 2014-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6
WO2007136989A2 (en) 2006-05-05 2007-11-29 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of dgat2
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs

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