CA2542835A1 - Inhibition a mediation d'interference d'arn de l'expression genetique a l'aide d'acide nucleique d'interference court (sina) - Google Patents
Inhibition a mediation d'interference d'arn de l'expression genetique a l'aide d'acide nucleique d'interference court (sina) Download PDFInfo
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- CA2542835A1 CA2542835A1 CA002542835A CA2542835A CA2542835A1 CA 2542835 A1 CA2542835 A1 CA 2542835A1 CA 002542835 A CA002542835 A CA 002542835A CA 2542835 A CA2542835 A CA 2542835A CA 2542835 A1 CA2542835 A1 CA 2542835A1
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- nucleotides
- sina
- sina molecule
- molecule
- rna
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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Applications Claiming Priority (19)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US10/693,059 US20080039414A1 (en) | 2002-02-20 | 2003-10-23 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US10/693,059 | 2003-10-23 | ||
| US10/720,448 | 2003-11-24 | ||
| US10/720,448 US8273866B2 (en) | 2002-02-20 | 2003-11-24 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA) |
| US10/727,780 | 2003-12-03 | ||
| US10/727,780 US20050233329A1 (en) | 2002-02-20 | 2003-12-03 | Inhibition of gene expression using duplex forming oligonucleotides |
| US10/757,803 | 2004-01-14 | ||
| US10/757,803 US20050020525A1 (en) | 2002-02-20 | 2004-01-14 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US54348004P | 2004-02-10 | 2004-02-10 | |
| US60/543,480 | 2004-02-10 | ||
| US10/780,447 | 2004-02-13 | ||
| US10/780,447 US7491805B2 (en) | 2001-05-18 | 2004-02-13 | Conjugates and compositions for cellular delivery |
| US10/826,966 US20050032733A1 (en) | 2001-05-18 | 2004-04-16 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA) |
| US10/826,966 | 2004-04-16 | ||
| USPCT/US04/13456 | 2004-04-30 | ||
| PCT/US2004/013456 WO2005041859A2 (fr) | 2003-04-30 | 2004-04-30 | Conjugues et compositions |
| USPCT/US04/16390 | 2004-05-24 | ||
| PCT/US2004/016390 WO2005019453A2 (fr) | 2001-05-18 | 2004-05-24 | Interference arn a mediation assuree par l'inhibition de genes au moyen de petit acide nucleique interferent (ansi) modifie chimiquement |
| PCT/US2004/027403 WO2005044981A2 (fr) | 2003-10-23 | 2004-08-20 | Inhibition a mediation d'interference d'arn de l'expression genetique a l'aide d'acide nucleique d'interference court (sina) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2542835A1 true CA2542835A1 (fr) | 2005-05-19 |
Family
ID=56290593
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002543013A Abandoned CA2543013A1 (fr) | 2003-10-23 | 2004-08-20 | Inhibition mediee par interference arn de l'expression genique de nogo et du recepteur nogo au moyen d'un petit acide nucleique interferent (sina) |
| CA002542835A Abandoned CA2542835A1 (fr) | 2003-10-23 | 2004-08-20 | Inhibition a mediation d'interference d'arn de l'expression genetique a l'aide d'acide nucleique d'interference court (sina) |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002543013A Abandoned CA2543013A1 (fr) | 2003-10-23 | 2004-08-20 | Inhibition mediee par interference arn de l'expression genique de nogo et du recepteur nogo au moyen d'un petit acide nucleique interferent (sina) |
Country Status (4)
| Country | Link |
|---|---|
| EP (3) | EP1675949A2 (fr) |
| JP (2) | JP2008506351A (fr) |
| CA (2) | CA2543013A1 (fr) |
| WO (3) | WO2005045035A2 (fr) |
Families Citing this family (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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) |
| WO2005066363A2 (fr) * | 2004-01-09 | 2005-07-21 | Medigen Biotechnology Corporation | Gene mg20 specifique du cancer |
| EP1704228B1 (fr) | 2004-01-16 | 2012-04-11 | Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. | Immunokinases |
| WO2006002538A1 (fr) | 2004-07-02 | 2006-01-12 | Protiva Biotherapeutics, Inc. | Molecules sirna immunostimulatrices et utilisations de celles-ci |
| CA2584921A1 (fr) * | 2004-10-22 | 2006-04-27 | Neuregenix Limited | Regeneration neuronale |
| WO2007013359A2 (fr) * | 2005-07-28 | 2007-02-01 | Oncotherapy Science, Inc. | Gene rasgef1a associe au cancer |
| EP2980220A1 (fr) | 2005-09-20 | 2016-02-03 | BASF Plant Science GmbH | Procédés améliorés de contrôle de l'expression de gènes |
| US20070099858A1 (en) * | 2005-10-03 | 2007-05-03 | Sirna Therapeutics, Inc. | RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA) |
| AU2013203219B2 (en) * | 2005-11-02 | 2016-09-22 | Arbutus Biopharma Corporation | MODIFIED siRNA MOLECULES AND USES THEREOF |
| US8101741B2 (en) | 2005-11-02 | 2012-01-24 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| WO2007056861A1 (fr) * | 2005-11-18 | 2007-05-24 | Protiva Biotherapeutics, Inc. | Attenuation de l'expression du gene du virus de la grippe par arnsi |
| EP1800695A1 (fr) * | 2005-12-21 | 2007-06-27 | Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. | Immuno-RNA conjugues |
| CA2913655A1 (fr) | 2006-01-27 | 2007-08-09 | Biogen Ma Inc. | Antagonistes des recepteurs nogo |
| GB2450840B (en) | 2006-05-11 | 2010-12-29 | Quark Pharmaceuticals Inc | Screening Systems Utilizing RTP801 |
| ES2874149T3 (es) * | 2006-05-11 | 2021-11-04 | Alnylam Pharmaceuticals Inc | Composiciones y métodos para inhibir la expresión del gen PCSK9 |
| EP2392583A1 (fr) * | 2006-05-19 | 2011-12-07 | Alnylam Europe AG. | Modulation ARNi de AHAS et utilisations thérapeutiques associées |
| CA2653052A1 (fr) * | 2006-05-19 | 2007-11-29 | The Scripps Research Institute | Traitement du mauvais repliement de proteines |
| EP2584051B1 (fr) | 2006-05-22 | 2014-07-16 | Alnylam Pharmaceuticals Inc. | Compositions et procédés d'inhibition de l'expressions du gène IKK-B |
| WO2007141796A2 (fr) | 2006-06-09 | 2007-12-13 | Quark Pharmaceuticals, Inc. | Utilisations thérapeutiques d'inhibiteurs de rtp801l |
| US7915399B2 (en) | 2006-06-09 | 2011-03-29 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| DE102006032424A1 (de) * | 2006-07-13 | 2008-01-17 | Ernst-Moritz-Arndt Universität Greifswald | Behandlung von T-Zell-Malignomen |
| EP2121925A2 (fr) * | 2007-03-02 | 2009-11-25 | MDRNA, Inc. | Composes d'acide nucleique pour inhiber l'expression du gene ras et utilisations de ceux-ci |
| US11078262B2 (en) | 2007-04-30 | 2021-08-03 | Allergan, Inc. | High viscosity macromolecular compositions for treating ocular conditions |
| FR2921935B1 (fr) * | 2007-10-05 | 2011-10-28 | Centre Nat Rech Scient | Procede de criblage de composes utilisables pour le traitement de troubles respiratoires |
| US8614311B2 (en) | 2007-12-12 | 2013-12-24 | Quark Pharmaceuticals, Inc. | RTP801L siRNA compounds and methods of use thereof |
| EP2123773A1 (fr) * | 2008-05-21 | 2009-11-25 | sanofi-aventis | Utilisation de modulateurs GPR151 pour le traitement de la douleur |
| EP2756845B1 (fr) * | 2009-04-03 | 2017-03-15 | Dicerna Pharmaceuticals, Inc. | Procédés et compositions pour l'inhibition spécifique de KRAS par de l'ARN double brin asymétrique |
| CN104651362A (zh) | 2009-04-03 | 2015-05-27 | 戴瑟纳制药公司 | 利用不对称双链rna特异性抑制kras的方法和组合物 |
| US20120128673A1 (en) | 2009-05-20 | 2012-05-24 | Schering Corporation | Modulation of pilr receptors to treat microbial infections |
| WO2011031600A1 (fr) | 2009-09-10 | 2011-03-17 | Schering Corporation | Utilisation d'antagonistes de l'il-33 à des fins de traitement des maladies fibrotiques |
| US8691227B2 (en) | 2009-12-17 | 2014-04-08 | Merck Sharp & Dohme Corp. | Methods of treating multiple sclerosis, rheumatoid arthritis and inflammatory bowel disease using agonists antibodies to PILR-α |
| WO2011127175A1 (fr) * | 2010-04-06 | 2011-10-13 | Isis Pharmaceuticals, Inc. | Modulation de l'expression de cd130 (gp130) |
| WO2011163499A2 (fr) * | 2010-06-23 | 2011-12-29 | Opko Curna, Llc | Traitement de maladies liées à la sous-unité alpha du canal sodique voltage-dépendant (scna) par inhibition du produit de transcription naturel antisens à la scna |
| WO2012027206A1 (fr) | 2010-08-24 | 2012-03-01 | Merck Sharp & Dohme Corp. | Agents à base d'arni à un seul brin contenant une séquence intercalaire interne ne correspondant pas à un acide nucléique |
| WO2012058210A1 (fr) | 2010-10-29 | 2012-05-03 | Merck Sharp & Dohme Corp. | INHIBITION FACILITÉE PAR L'INTERFÉRENCE D'ARN DE L'EXPRESSION D'UN GÈNE AU MOYEN D'ACIDES NUCLÉIQUES INTERFÉRENTS COURTS (siNA) |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP3260540A1 (fr) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Arn non codants associés à polycomb |
| WO2013070771A1 (fr) * | 2011-11-07 | 2013-05-16 | Isis Pharmaceuticals, Inc. | Administration d'oligonucléotides anti-sens du facteur xi |
| EP3272868A1 (fr) * | 2012-05-02 | 2018-01-24 | Arrowhead Pharmaceuticals, Inc. | Compositions organiques pour traiter des maladies associées à kras |
| AU2013259387B2 (en) * | 2012-05-09 | 2016-12-15 | Strike Bio, Inc. | Bi-functional short-hairpin rna (bi-shrna) specific for single-nucleotide kras mutations |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Smn遺伝子ファミリー発現を調節するための組成物及び方法 |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| CA2873769A1 (fr) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions et methodes pour moduler l'expression de la famille multigenique de l'hemoglobine |
| CA2873766A1 (fr) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions et methodes pour moduler l'expression de atp2a2 |
| BR112014028634A2 (pt) | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| US10125369B2 (en) | 2012-12-05 | 2018-11-13 | Alnylam Pharmaceuticals, Inc. | PCSK9 iRNA compositions and methods of use thereof |
| CA2966044A1 (fr) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Procedes de modulation de la repression genique dependant d'atrx |
| WO2016149455A2 (fr) | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | Interactome arn de complexe répressif polycomb 1 (prc1) |
| EP3328408B1 (fr) * | 2015-07-31 | 2020-10-07 | Vascular Biogenics Ltd. | Protéine 2 contenant le domaine du sperme motile et inflammation |
| CN118697894A (zh) | 2015-08-25 | 2024-09-27 | 阿尔尼拉姆医药品有限公司 | 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物 |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| CN106755293B (zh) * | 2016-09-23 | 2020-09-04 | 宁波大学 | 一种与肺癌辅助诊断相关的lncRNA标志物及其应用 |
| EP4035659A1 (fr) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes destinés à l'administration d'agents thérapeutiques |
| CN108251529B (zh) * | 2018-02-01 | 2021-06-01 | 新乡医学院 | Ano5在制备治疗胰腺癌的药物中的应用 |
| CN110564771B (zh) * | 2018-06-05 | 2022-09-13 | 中国科学院脑科学与智能技术卓越创新中心 | 脑钙化病模型的制备方法 |
| CN108815512B (zh) * | 2018-08-23 | 2021-06-11 | 河南师范大学 | 一种抗癌药物及其应用 |
| CN112553333B (zh) * | 2020-12-08 | 2022-03-08 | 南方医科大学深圳医院 | miR-1207及其靶基因在检测喉鳞癌中的应用 |
| US20250304966A1 (en) | 2020-12-23 | 2025-10-02 | Argonaute RNA Limited | Treatment of cardiovascular disease |
| WO2023041508A2 (fr) | 2021-09-14 | 2023-03-23 | Argonaute RNA Limited | Traitement d'une maladie cardiovasculaire |
| US20250333732A1 (en) * | 2022-05-23 | 2025-10-30 | Empirico Inc. | Treatment of sos2 related diseases and disorders |
| CN120769911A (zh) * | 2023-02-22 | 2025-10-10 | 艾尔塔米拉治疗公司 | 用于治疗疾病的抑制kras的组合物和方法 |
| WO2025129117A1 (fr) * | 2023-12-15 | 2025-06-19 | Ivy Natal Inc. | Compositions et procédés pour induire une méiose |
| WO2025235822A1 (fr) * | 2024-05-10 | 2025-11-13 | Gmp Biotechnology Limited | Agents thérapeutiques contre le cancer utilisant tgfb2 et tgm6 |
| CN119530226B (zh) * | 2024-11-28 | 2025-07-25 | 河北农业大学 | 一种抑制鸡lnc-CA13基因表达的shRNA、重组载体、重组菌及其应用 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003070918A2 (fr) * | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Inhibition mediee par interference arn d'une expression genique faisant appel a des acides nucleiques interferants courts chimiquement modifies (sina) |
| WO2002081628A2 (fr) * | 2001-04-05 | 2002-10-17 | Ribozyme Pharmaceuticals, Incorporated | Modulation de l'expression genique associee a la proliferation inflammatoire et a la croissance de neurites, par des procedes faisant intervenir l'acide nucleique |
| WO2001059103A2 (fr) * | 2000-02-11 | 2001-08-16 | Ribozyme Pharmaceuticals, Inc. | Methode et reactif destines a la modulation et au diagnostic de l'expression genetique de cd20 et de nogo |
| US20040259247A1 (en) * | 2000-12-01 | 2004-12-23 | Thomas Tuschl | Rna interference mediating small rna molecules |
| WO2002097114A2 (fr) * | 2001-05-29 | 2002-12-05 | Sirna Therapeutics, Inc. | Traitement a l'acide nucleique de maladies ou d'affections associees aux taux de ras, her2 et hiv |
| WO2003070896A2 (fr) * | 2002-02-20 | 2003-08-28 | Sirna Therapeutics, Inc | Inhibition, induite par adn d'interference, de l'expression genique d'un antigene nucleaire de proliferation cellulaire (pcna) a l'aide d'un acide nucleique a interference courte (sina) |
| WO2003070887A2 (fr) * | 2002-02-20 | 2003-08-28 | Sirna Therapeutics, Inc. | Inhibition mediee par interference d'arn de l'expression du gene de la proteine ezh2 du groupe polycomb a l'aide d'un acide nucleique court interferent (sina) |
| AU2003207708A1 (en) * | 2002-02-20 | 2003-09-09 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of map kinase genes |
-
2004
- 2004-08-20 WO PCT/US2004/026930 patent/WO2005045035A2/fr not_active Ceased
- 2004-08-20 JP JP2006536613A patent/JP2008506351A/ja not_active Withdrawn
- 2004-08-20 CA CA002543013A patent/CA2543013A1/fr not_active Abandoned
- 2004-08-20 EP EP04781588A patent/EP1675949A2/fr not_active Withdrawn
- 2004-08-20 EP EP04781982A patent/EP1682661A2/fr not_active Withdrawn
- 2004-08-20 WO PCT/US2004/027403 patent/WO2005044981A2/fr not_active Ceased
- 2004-08-20 EP EP04816819A patent/EP1675953A2/fr not_active Withdrawn
- 2004-08-20 WO PCT/US2004/027333 patent/WO2005040379A2/fr not_active Ceased
- 2004-08-20 CA CA002542835A patent/CA2542835A1/fr not_active Abandoned
- 2004-08-20 JP JP2006536609A patent/JP2007522793A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2005040379A3 (fr) | 2006-03-02 |
| JP2008506351A (ja) | 2008-03-06 |
| CA2543013A1 (fr) | 2005-05-19 |
| EP1675953A2 (fr) | 2006-07-05 |
| EP1675949A2 (fr) | 2006-07-05 |
| WO2005040379A2 (fr) | 2005-05-06 |
| WO2005044981A2 (fr) | 2005-05-19 |
| WO2005045035A3 (fr) | 2005-12-08 |
| WO2005044981A3 (fr) | 2005-09-22 |
| JP2007522793A (ja) | 2007-08-16 |
| EP1682661A2 (fr) | 2006-07-26 |
| WO2005045035A2 (fr) | 2005-05-19 |
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