JP2004512810A - 核酸に基づく遺伝子発現の調節剤 - Google Patents

核酸に基づく遺伝子発現の調節剤 Download PDF

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JP2004512810A
JP2004512810A JP2001520859A JP2001520859A JP2004512810A JP 2004512810 A JP2004512810 A JP 2004512810A JP 2001520859 A JP2001520859 A JP 2001520859A JP 2001520859 A JP2001520859 A JP 2001520859A JP 2004512810 A JP2004512810 A JP 2004512810A
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nucleic acid
acid molecule
enzymatic nucleic
rna
enzymatic
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JP2004512810A5 (enExample
Inventor
マクスウィゲン，ジェイムズ
ウスマン，ナシム
ブラット，ローレンス
ベイジェルマン，レオニド
バージン，アレックス
カーペイスキー，アレクサンダー
マチュリックーアダミック，ジャセンカ
スウィードラー，デイビッド
ドレイパー，ケネス
チョーリラ，バラート
スティンチコーム，ダン
ビュードリー，アンバー
ジネン，ショーン
ラグウィッグ，ジャノス
スプロート，ブライアン・エス
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Sirna Therapeutics Inc
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Sirna Therapeutics Inc
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Priority claimed from US09/474,432 external-priority patent/US6528640B1/en
Priority claimed from US09/476,387 external-priority patent/US6617438B1/en
Application filed by Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Publication of JP2004512810A publication Critical patent/JP2004512810A/ja
Publication of JP2004512810A5 publication Critical patent/JP2004512810A5/ja
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JP2001520859A 1999-08-31 2000-08-30 核酸に基づく遺伝子発現の調節剤 Pending JP2004512810A (ja)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US15171399P 1999-08-31 1999-08-31
US15623699P 1999-09-27 1999-09-27
US15646799P 1999-09-27 1999-09-27
US40664399A 1999-09-27 1999-09-27
US43643099A 1999-11-08 1999-11-08
US16910099P 1999-12-06 1999-12-06
US17361299P 1999-12-29 1999-12-29
US09/474,432 US6528640B1 (en) 1997-11-05 1999-12-29 Synthetic ribonucleic acids with RNAse activity
US09/476,387 US6617438B1 (en) 1997-11-05 1999-12-30 Oligoribonucleotides with enzymatic activity
US49882400A 2000-02-04 2000-02-04
US53102500A 2000-03-20 2000-03-20
US19776900P 2000-04-14 2000-04-14
US57822300A 2000-05-23 2000-05-23
US63638500A 2000-08-09 2000-08-09
PCT/US2000/023998 WO2001016312A2 (en) 1999-08-31 2000-08-30 Nucleic acid based modulators of gene expression

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JP2004512810A true JP2004512810A (ja) 2004-04-30
JP2004512810A5 JP2004512810A5 (enExample) 2005-01-06

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EP (1) EP1212416A2 (enExample)
JP (1) JP2004512810A (enExample)
CA (1) CA2403243A1 (enExample)
WO (1) WO2001016312A2 (enExample)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4837555B2 (ja) * 2003-02-28 2011-12-14 アイシス ファーマシューティカルズ， インコーポレーテッド 成長ホルモン受容体の発現およびインスリン様成長因子の発現のモジュレーション
JP2013544251A (ja) * 2010-11-15 2013-12-12 ラモット・アット・テル・アビブ・ユニバーシテイ・リミテッド アミロイド線維形成関連状態を処置するためのジペプチドアナログ
JP2015528013A (ja) * 2012-07-27 2015-09-24 ビアル−ポルテラ エ コンパニア，ソシエダッド アノニマ 置換ウレア化合物の合成方法
WO2015173208A3 (en) * 2014-05-15 2016-02-18 F. Hoffmann-La Roche Ag Oligomers and oligomer conjugates
JP2024506351A (ja) * 2021-02-11 2024-02-13 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 Ｐｌｎの発現を低減するための化合物及び方法
US12378559B2 (en) 2018-11-02 2025-08-05 University Of Essex Enterprises Limited Enzymatic nucleic acid molecules

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6482932B1 (en) * 1997-11-05 2002-11-19 Ribozyme Pharmaceuticals, Incorporated Nucleoside triphosphates and their incorporation into oligonucleotides
US6528640B1 (en) * 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US6617438B1 (en) * 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US20020055479A1 (en) * 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US7179796B2 (en) 2000-01-18 2007-02-20 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US7125660B2 (en) 2000-09-13 2006-10-24 Archemix Corp. Nucleic acid sensor molecules and methods of using same
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
WO2003070983A1 (en) * 2002-02-20 2003-08-28 Sirna Therapeutics, Inc RNA INTERFERENCE MEDIATED INHIBITION OF PROTEIN KINASE C ALPHA (PKC-ALPHA) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2002097114A2 (en) * 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Nucleic acid treatment of diseases or conditions related to levels of ras, her2 and hiv
WO2003034985A2 (en) * 2001-10-22 2003-05-01 University Of Rochester Telomerase interference
AU2003215161A1 (en) * 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF TELOMERASE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
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)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
ATE443776T1 (de) * 2002-05-17 2009-10-15 Us Gov Health & Human Serv Molekulare identifizierung von aspergillus- spezies
US7507808B2 (en) 2002-12-12 2009-03-24 Isis Pharmaceuticals, Inc. Modulation of endothelial lipase expression
US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
FR2848573B1 (fr) * 2002-12-13 2005-04-08 Exonhit Therapeutics Sa Compositions et methodes pour la detection et le traitement de pathologies neurodegeneratives
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
US7846906B2 (en) 2003-02-28 2010-12-07 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
AU2005233583B2 (en) * 2004-04-08 2011-02-03 Sangamo Therapeutics, Inc. Methods and compositions for modulating cardiac contractility
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20090010907A1 (en) * 2004-12-14 2009-01-08 National Institute Of Immunology Dnazymes for Inhibition of Japanese Encephalitis Virus Replication
AU2006222563A1 (en) 2005-03-08 2006-09-14 Biota Scientific Management Pty Ltd. Bicyclic nucleosides and nucleotides as therapeutic agents
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
JP2010500011A (ja) 2006-08-08 2010-01-07 ガンサー ハートマン ５’リン酸オリゴヌクレオチドの構造および使用
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US9738680B2 (en) 2008-05-21 2017-08-22 Rheinische Friedrich-Wilhelms-Universität Bonn 5′ triphosphate oligonucleotide with blunt end and uses thereof
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
ES2651308T3 (es) 2009-10-16 2018-01-25 Glaxo Group Limited Inhibidores antisentido de HBV
JP6106085B2 (ja) 2010-08-24 2017-03-29 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 内部非核酸スペーサーを含む一本鎖ＲＮＡｉ剤
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
CN105886506A (zh) 2011-04-13 2016-08-24 Isis制药公司 Ptp1b 表达的反义调节
SI3505528T1 (sl) 2011-04-21 2021-04-30 Glaxo Group Limited Modulacija izražanja virusa hepatitisa B (HBV)
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
DE102015113038B3 (de) * 2015-07-13 2017-01-19 Simone Kann Oligonucleotide und deren Verwendung
WO2017048620A1 (en) * 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
US11634716B2 (en) 2017-10-16 2023-04-25 University of Pittsburgh—of the Commonwealth System of Higher Education Genetically modified mesenchymal stem cells for use in cardiovascular prosthetics
KR20260021596A (ko) 2023-04-05 2026-02-13 아이오니스 파마수티컬즈, 인코포레이티드 Pln 발현을 감소시키는 화합물 및 방법
AU2024308906A1 (en) * 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
US20260159836A1 (en) * 2024-12-05 2026-06-11 Soufflé Therapeutics, Inc. Oligonucleotide-mediated knockdown of pln

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU1430097A (en) * 1996-01-16 1997-08-11 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
AU721758B2 (en) * 1996-04-02 2000-07-13 Gene Shears Pty. Limited Asymmetric hammerhead ribozymes
AU735522C (en) * 1997-04-29 2005-04-07 Scripps Research Institute, The Enzymatic dna molecules

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US9630989B2 (en) 2010-11-15 2017-04-25 Ramot At Tel-Aviv University Ltd. Dipeptide analogs for treating conditions associated with amyloid fibril formation
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US10421967B2 (en) 2014-05-15 2019-09-24 Hoffmann-La Roche Inc. Oligomers and oligomer conjugates
WO2015173208A3 (en) * 2014-05-15 2016-02-18 F. Hoffmann-La Roche Ag Oligomers and oligomer conjugates
EA034924B1 (ru) * 2014-05-15 2020-04-07 Ф. Хоффманн-Ля Рош Аг Олигомеры и олигомерные конъюгаты
US10767181B2 (en) 2014-05-15 2020-09-08 Hoffmann-La Roche Inc. Oligomers and oligomer conjugates
US11591598B2 (en) 2014-05-15 2023-02-28 Hoffmann-La Roche Inc. Oligomers and oligomer conjugates
US12281307B2 (en) 2014-05-15 2025-04-22 Hoffmann-La Roche Inc. Oligomers and oligomer conjugates
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JP2024506351A (ja) * 2021-02-11 2024-02-13 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 Ｐｌｎの発現を低減するための化合物及び方法

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