ES2790574T3 - Modulación de expresión de prekallikrein (PKK) - Google Patents

Modulación de expresión de prekallikrein (PKK) Download PDF

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Publication number
ES2790574T3
ES2790574T3 ES14839032T ES14839032T ES2790574T3 ES 2790574 T3 ES2790574 T3 ES 2790574T3 ES 14839032 T ES14839032 T ES 14839032T ES 14839032 T ES14839032 T ES 14839032T ES 2790574 T3 ES2790574 T3 ES 2790574T3
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Prior art keywords
certain embodiments
pkk
antisense
nucleic acid
seq
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ES14839032T
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English (en)
Spanish (es)
Inventor
Susan Freier
Huynh-Hoa Bui
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • C12N15/1137—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 enzymes
    • 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
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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    • A61P7/10—Antioedematous agents; Diuretics
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • 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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    • 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
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21034—Plasma kallikrein (3.4.21.34)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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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
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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  • Proteomics, Peptides & Aminoacids (AREA)
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ES14839032T 2013-08-28 2014-08-28 Modulación de expresión de prekallikrein (PKK) Active ES2790574T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361871175P 2013-08-28 2013-08-28
PCT/US2014/053266 WO2015031679A2 (en) 2013-08-28 2014-08-28 Modulation of prekallikrein (pkk) expression

Publications (1)

Publication Number Publication Date
ES2790574T3 true ES2790574T3 (es) 2020-10-28

Family

ID=52587489

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ES14839032T Active ES2790574T3 (es) 2013-08-28 2014-08-28 Modulación de expresión de prekallikrein (PKK)

Country Status (14)

Country Link
US (5) US9670492B2 (enExample)
EP (2) EP3038627B1 (enExample)
JP (2) JP6652922B2 (enExample)
KR (1) KR102365486B1 (enExample)
CN (1) CN105517556B (enExample)
AU (2) AU2014312196C1 (enExample)
BR (1) BR112016004093A2 (enExample)
CA (1) CA2921839A1 (enExample)
ES (1) ES2790574T3 (enExample)
IL (1) IL244047A0 (enExample)
MX (1) MX2016002587A (enExample)
NZ (1) NZ716816A (enExample)
RU (1) RU2712559C9 (enExample)
WO (1) WO2015031679A2 (enExample)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9670492B2 (en) 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
HUE052709T2 (hu) * 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
KR102569559B1 (ko) * 2016-09-16 2023-08-23 다케다 파머수티컬 컴패니 리미티드 유전성 혈관부종에 대한 rna 바이오마커
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
CN118291456A (zh) 2017-12-01 2024-07-05 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
JP7360716B2 (ja) 2017-12-01 2023-10-13 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物および複合体ならびに調製方法と使用
WO2019105414A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
HRP20250277T1 (hr) 2017-12-01 2025-05-09 Suzhou Ribo Life Science Co., Ltd. Dvolančani oligonukleotid, sastav i konjugat koji sadrži dvolančani oligonukleotid, postupak njihove pripreme i upotreba
CN110944675B9 (zh) 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110959011B (zh) 2017-12-29 2023-03-28 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
JP7376952B2 (ja) 2018-09-30 2023-11-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド ｓｉＲＮＡ複合体及びその調製方法と使用
JP7507495B2 (ja) 2018-12-28 2024-06-28 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
JP7610848B2 (ja) 2019-05-22 2025-01-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
CN113286888B (zh) 2019-05-22 2023-12-22 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113227376B (zh) 2019-05-22 2024-04-09 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113891939B (zh) * 2019-05-24 2024-04-02 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN115362256A (zh) 2020-02-07 2022-11-18 因特利亚治疗公司 用于激肽释放酶（klkb1）基因编辑的组合物和方法
US20230149438A1 (en) * 2020-03-13 2023-05-18 Ionis Pharmaceuticals, Inc. Compositions and methods for treating and preventing prekallikrein-associated conditions
WO2021248027A1 (en) * 2020-06-05 2021-12-09 Ionis Pharmaceuticals, Inc. Compositions and methods of inhibiting angiotensin-converting enzyme 2 (ace2) and transmembrane serine protease 2 (tmprss2)
KR20230108728A (ko) 2020-11-18 2023-07-18 아이오니스 파마수티컬즈, 인코포레이티드 앤지오텐시노겐 발현을 조절하기 위한 화합물 및 방법
EP4273245A4 (en) * 2020-12-29 2024-12-11 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE WITH THE NUCLEIC ACID, MANUFACTURING PROCESS THEREOF AND USE THEREOF
US11833221B2 (en) * 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
AU2022357561A1 (en) 2021-10-01 2024-04-18 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
TW202346586A (zh) * 2022-01-28 2023-12-01 大陸商上海舶望製藥有限公司 用於抑制前激肽釋放酶（pkk）蛋白表達的組合物和方法

Family Cites Families (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2699508A (en) 1951-12-21 1955-01-11 Selectronics Inc Method of mounting and construction of mounting for low frequency piezoelectric crystals
DE2654124A1 (de) 1976-11-29 1978-06-01 Bayer Ag Derivate des trypsin-kallikrein-inhibitors aus rinderorganen (bpti) mit proteasenhemmwirkung und antiphlogistischer wirkung, ihre herstellung und ihre verwendung als arzneimittel
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
FR2599752B1 (fr) 1986-06-10 1989-11-03 Transgene Sa Variants de l'alpha1- antitrypsine utiles notamment comme inhibiteurs de la kallikreine
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
EP0455905B1 (en) 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
ATE515510T1 (de) * 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
WO1994014226A1 (en) 1992-12-14 1994-06-23 Honeywell Inc. Motor system with individually controlled redundant windings
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US6057287A (en) 1994-01-11 2000-05-02 Dyax Corp. Kallikrein-binding "Kunitz domain" proteins and analogues thereof
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5681940A (en) 1994-11-02 1997-10-28 Icn Pharmaceuticals Sugar modified nucleosides and oligonucleotides
US5786328A (en) 1995-06-05 1998-07-28 Genentech, Inc. Use of kunitz type plasma kallikrein inhibitors
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US5869637A (en) 1996-07-22 1999-02-09 Incyte Pharmaceuticals, Inc. Human Kallikrein
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
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
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
DE19935303A1 (de) * 1999-07-28 2001-02-08 Aventis Pharma Gmbh Oligonukleotide zur Inhibierung der Expression von humanem eg5
US20020082227A1 (en) 1999-09-30 2002-06-27 Scott Henry Use of oligonucleotides for inhibition of complement activation
JP2003519231A (ja) 1999-12-30 2003-06-17 カー・ユ・ルーベン・リサーチ・アンド・ディベロップメント シクロヘキセン核酸
CN100523215C (zh) * 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20040097441A1 (en) * 2002-11-16 2004-05-20 Isis Pharmaceuticals Inc. Modulation of NIMA-related kinase 6 expression
PT2298278E (pt) 2002-06-07 2016-02-25 Dyax Corp Prevenção e redução da perda de sangue e resposta inflamatória
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1730158A2 (en) 2003-08-06 2006-12-13 Cygene, Inc. Methods and compositions for in vitro and in vivo use of parallel stranded hairpins and triplex structures as nucleic acid ligands
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
WO2005022146A2 (en) 2003-08-30 2005-03-10 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with kallikrein 13 (klk13)
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
US20050118625A1 (en) 2003-10-02 2005-06-02 Mounts William M. Nucleic acid arrays for detecting gene expression associated with human osteoarthritis and human proteases
EP1713929A2 (en) 2004-02-03 2006-10-25 Bayer HealthCare AG Diagnostics and therapeutics for diseases associated with plasma kallikrein (klkb1)
US20050221354A1 (en) 2004-02-18 2005-10-06 Wyeth Nucleic acid arrays for monitoring expression profiles of drug target genes
WO2005083110A1 (en) 2004-02-26 2005-09-09 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with kallikrein 4 (klk4)
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
WO2005116265A2 (en) 2004-05-26 2005-12-08 Wyeth Probe arrays for expression profiling of rat genes
WO2006008002A2 (en) 2004-07-23 2006-01-26 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with kallikrein 1 (klk1)
US7235530B2 (en) 2004-09-27 2007-06-26 Dyax Corporation Kallikrein inhibitors and anti-thrombolytic agents and uses thereof
WO2006047842A2 (en) 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
US20060185027A1 (en) 2004-12-23 2006-08-17 David Bartel Systems and methods for identifying miRNA targets and for altering miRNA and target expression
WO2006091459A2 (en) 2005-02-24 2006-08-31 Joslin Diabetes Center, Inc. Compositions and methods for treating vascular permeability
JP4041154B2 (ja) 2005-05-13 2008-01-30 松下電器産業株式会社 混合音分離装置
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2051707B1 (en) 2006-07-31 2013-07-17 Activesite Pharmaceuticals, Inc. Inhibitors of plasma kallikrein
EP2081949B1 (en) 2006-09-22 2014-12-10 GE Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 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
WO2009026334A2 (en) 2007-08-21 2009-02-26 Genzyme Corporation Treatment with kallikrein inhibitors
WO2009026539A1 (en) 2007-08-23 2009-02-26 Genzyme Corporation Treatment with kallikrein inhibitors
US8095862B2 (en) 2007-10-10 2012-01-10 International Business Machines Corporation End-to-end cyclic redundancy check protection for high integrity fiber transfers
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
JP5409658B2 (ja) * 2008-03-13 2014-02-05 セレラ コーポレーション 静脈血栓症に関連した遺伝子多型、その検出方法および使用
JP5288456B2 (ja) * 2008-08-08 2013-09-11 富士フイルム株式会社 口腔扁平上皮癌の検出方法
WO2010036696A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
JP2013516389A (ja) 2009-01-06 2013-05-13 ダイアックス コーポレーション カリクレイン阻害剤による粘膜炎治療
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US8609866B2 (en) 2009-12-18 2013-12-17 Activesite Pharmaceuticals, Inc. Prodrugs of inhibitors of plasma kallikrein
ES2905545T3 (es) 2010-01-06 2022-04-11 Takeda Pharmaceuticals Co Proteínas de unión a calicreína plasmática
WO2012170945A2 (en) * 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
US9187749B2 (en) 2011-06-10 2015-11-17 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
AU2012275096B2 (en) 2011-06-29 2016-02-04 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (KLKB1) expression
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
TWI595885B (zh) 2012-05-02 2017-08-21 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
US20150141497A1 (en) 2012-06-15 2015-05-21 Joslin Diabetes Center, Inc. Methods for modulating kallikrein (klkb1) expression
US9670492B2 (en) 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
KR20160083876A (ko) 2013-11-14 2016-07-12 로슈 이노베이션 센터 코펜하겐 에이/에스 ApoB 안티센스 접합체 화합물
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
US20230149438A1 (en) 2020-03-13 2023-05-18 Ionis Pharmaceuticals, Inc. Compositions and methods for treating and preventing prekallikrein-associated conditions

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