MA28029A1 - Double helice d'arn d'interference possedant des extremites franches et des modifications en 3' - Google Patents

Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'

Info

Publication number
MA28029A1
MA28029A1 MA28861A MA28861A MA28029A1 MA 28029 A1 MA28029 A1 MA 28029A1 MA 28861 A MA28861 A MA 28861A MA 28861 A MA28861 A MA 28861A MA 28029 A1 MA28029 A1 MA 28029A1
Authority
MA
Morocco
Prior art keywords
aryl
alkyl
propeller
changes
double
Prior art date
Application number
MA28861A
Other languages
English (en)
Inventor
Jan Weiler
Jonathan Hall
Jean-Charles Bologna
Francois Jean-Charles Natt
Robert Haener
Original Assignee
Novartis Ag
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of MA28029A1 publication Critical patent/MA28029A1/fr

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/311—Phosphotriesters
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Apparatus For Disinfection Or Sterilisation (AREA)

Abstract

La présente invention porte sur des composés d'ARN double brin pourvus d'au moins une extrémité franche comprenant au moins une extrémité 3' de formule (I). dans laquelle X représente O ou S; R1 et R2 représentent, indépendamment, OH, NH2, SH, alkyle, aryle, alkyl-aryle, aryl-alkyle, où alkyle, aryle, alkyl-aryle, aryl-alkyle peuvent être substitués par des hétéro-atomes additionnels et des groupes fonctionnels; de préférence, un hétéro-atome est sélectionné dans le groupe de N, O ou S ou un groupe fonctionnel est sélectionné dans le groupe OH, NH2, SH, l'acide carboxylique ou un ester; ou R| et R2 peuvent être de la formule Y-Z dans laquelle Y représente O. N, S et Z représente H, alkyle, aryle, alkyl-aryle, aryl-alkyle, où alkyle, aryle, alkyl-aryle, aryl-alkyle peuvent être substitués par des hétéro-atomes additionnels, de préférence un hétéro-atome sélectionné dans le groupe de N, O ou S. Dans la formule de cette invention, l'ARN double brin induit une interférence ARN. Les modifications préférées à l'extrémité 3' sont: phosphate, phosphorothioate, ribonucléoside abasique. hydroxypropyle, phosphodiester.
MA28861A 2003-08-28 2006-03-08 Double helice d'arn d'interference possedant des extremites franches et des modifications en 3' MA28029A1 (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US49851403P 2003-08-28 2003-08-28

Publications (1)

Publication Number Publication Date
MA28029A1 true MA28029A1 (fr) 2006-07-03

Family

ID=34272687

Family Applications (1)

Application Number Title Priority Date Filing Date
MA28861A MA28029A1 (fr) 2003-08-28 2006-03-08 Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'

Country Status (21)

Country Link
US (2) US20070203084A1 (fr)
EP (3) EP1660657A1 (fr)
JP (2) JP2007503803A (fr)
KR (3) KR101059315B1 (fr)
CN (2) CN1845993B (fr)
AU (1) AU2004269150C1 (fr)
BR (1) BRPI0413146A (fr)
CA (1) CA2536333C (fr)
CO (1) CO5680494A2 (fr)
EC (1) ECSP066388A (fr)
IL (1) IL173565A (fr)
IS (1) IS8358A (fr)
MA (1) MA28029A1 (fr)
MX (1) MXPA06002216A (fr)
NO (1) NO20061345L (fr)
NZ (1) NZ545360A (fr)
RU (2) RU2399671C2 (fr)
SG (1) SG152279A1 (fr)
TN (1) TNSN06065A1 (fr)
WO (1) WO2005021749A1 (fr)
ZA (1) ZA200601003B (fr)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1660657A1 (fr) 2003-08-28 2006-05-31 Novartis AG Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'
GB0608838D0 (en) * 2006-05-04 2006-06-14 Novartis Ag Organic compounds
CA2666657A1 (fr) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Molecules d'acide nucleique ouvertes ou coupees et leurs utilisations
JP4900943B2 (ja) * 2006-12-25 2012-03-21 独立行政法人産業技術総合研究所 ヌクレアーゼ耐性及びｒｎａ干渉効果に優れた修飾型二本鎖ｒｎａ
EP2316943B1 (fr) 2007-07-05 2013-06-19 Novartis AG ARNDB pour le traitement de l'infection virale
EP2260102A1 (fr) 2008-03-25 2010-12-15 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Traitement du cancer par régulation à la baisse de frizzled-4 et/ou frizzled-1
WO2010100247A1 (fr) 2009-03-06 2010-09-10 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research Nouvelle thérapie contre l'anxiété
EP2241323A1 (fr) 2009-04-14 2010-10-20 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Tenascine-W et cancers du cerveau
EP2292266A1 (fr) 2009-08-27 2011-03-09 Novartis Forschungsstiftung, Zweigniederlassung Traitement du cancer en modulant la copine III
US20120244170A1 (en) 2009-09-22 2012-09-27 Rafal Ciosk Treating cancer by modulating mex-3
WO2011045352A2 (fr) 2009-10-15 2011-04-21 Novartis Forschungsstiftung Tyrosine kinase de la rate et cancers du cerveau
WO2011072082A2 (fr) 2009-12-09 2011-06-16 Nitto Denko Corporation Modulation de l'expression de hsp47
EP3000885B1 (fr) 2009-12-18 2018-07-25 Arrowhead Pharmaceuticals, Inc. Compositions organiques de traitement des pathologies liées à hsf1
CA3009891C (fr) 2009-12-23 2020-09-15 Novartis Ag Lipides, compositions lipidiques, et procedes d'utilisation associes
US20120296403A1 (en) 2010-02-10 2012-11-22 Novartis Ag Methods and compounds for muscle growth
WO2011107586A1 (fr) 2010-03-05 2011-09-09 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research, Smoc1, ténascine-c et cancers du cerveau
EP2561076A1 (fr) 2010-04-19 2013-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Modulation de xrn1
US10913767B2 (en) * 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
DK2561077T3 (en) 2010-04-23 2016-08-01 Arrowhead Res Corp Organic compositions for the treatment of beta-ENaC-related diseases
US20130089538A1 (en) 2010-06-10 2013-04-11 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute forBiomedical Researh Treating cancer by modulating mammalian sterile 20-like kinase 3
US9181553B2 (en) 2011-06-06 2015-11-10 Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Method of treatment of breast cancers over-expressing the SHP2 signature genes
CN103687951B (zh) * 2011-07-18 2019-08-02 德福根有限公司 对昆虫害虫具有抗性的植物
CA3185394A1 (fr) 2011-09-02 2013-03-07 Arrowhead Pharmaceuticals, Inc. Compositions organiques de traitement de maladies associees a hsf1
US20140314787A1 (en) 2011-11-08 2014-10-23 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute Treatment for neurodegenerative diseases
US20140294732A1 (en) 2011-11-08 2014-10-02 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute Early diagnostic of neurodegenerative diseases
US20140343128A1 (en) 2011-11-15 2014-11-20 Novartis Ag Combination of a phosphoinositide 3-kinase inhibitor and a modulator of the Janus Kinase 2 - Signal Transducer and Activator of Transcription 5 pathway
WO2013102825A1 (fr) 2012-01-02 2013-07-11 Novartis Ag Cdcp1 et cancer du sein
EP2802658A2 (fr) 2012-01-09 2014-11-19 Novartis AG Compositions organiques pour traiter des maladies associées à la bêta-caténine
US20150266961A1 (en) 2012-03-29 2015-09-24 Novartis Forschungsstiftung, Zweigniederlassung, Fridrich Miescher Institute Inhibition of interleukin-8 and/or its receptor cxcr1 in the treatment of her2/her3-overexpressing breast cancer
EA201492004A1 (ru) 2012-05-02 2015-08-31 Новартис Аг Органические композиции для лечения kras-ассоциированных заболеваний
KR101567576B1 (ko) * 2012-05-22 2015-11-10 올릭스 주식회사 세포 내 관통능을 가지고 ｒｎａ 간섭을 유도하는 핵산 분자 및 그 용도
US20150218238A1 (en) 2012-06-29 2015-08-06 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Resear Treating diseases by modulating a specific isoform of mkl1
WO2014006114A1 (fr) 2012-07-05 2014-01-09 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Nouveau traitement pour maladies neurodégénératives
US20150224190A1 (en) 2012-07-06 2015-08-13 Mohamed Bentires-Alj Combination of a phosphoinositide 3-kinase inhibitor and an inhibitor of the IL-8/CXCR interaction
CN105247051A (zh) 2013-02-28 2016-01-13 箭头研究公司 治疗epas1相关疾病的有机组合物
KR102144777B1 (ko) * 2013-06-19 2020-08-18 제이더블유중외제약 주식회사 결정형 메로페넴 삼수화물의 제조방법
ES2761587T3 (es) 2013-08-07 2020-05-20 Friedrich Miescher Institute For Biomedical Res Nuevo método de cribado para el tratamiento de la ataxia de Friedreich
WO2015051135A2 (fr) 2013-10-04 2015-04-09 Novartis Ag Compositions organiques destinées au traitement de maladies associées à l'hepcidine
HK1221912A1 (zh) 2013-10-04 2017-06-16 Novartis Ag 用於治疗乙肝病毒的有机化合物
WO2015051044A2 (fr) 2013-10-04 2015-04-09 Novartis Ag Formats inédits pour composés organiques utiisables en interférence arn
EP3052464B1 (fr) 2013-10-04 2020-04-15 Novartis AG 3'end caps pour des agents arni utilisés dans l'interférence d'arn
WO2015051366A2 (fr) 2013-10-04 2015-04-09 Novartis Ag Formats inédits pour composés organiques utilisables en interférence arn
KR101696704B1 (ko) * 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
US20170137824A1 (en) 2014-06-13 2017-05-18 Indranil BANERJEE New treatment against influenza virus
EP3164129A1 (fr) 2014-07-01 2017-05-10 Friedrich Miescher Institute for Biomedical Research Combinaison d'un inhibiteur de braf v600e et d'un inhibiteur de mertk pour le traitement du mélanome
EP3736334A1 (fr) 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Compositions d'arni pour le traitement de pathologies liées à l'apoc3
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
EP3191592A1 (fr) 2014-09-11 2017-07-19 Novartis AG Inhibition de prmt5 dans le traitement de maladies liées à une déficience en mtap
EP3197557A1 (fr) 2014-09-24 2017-08-02 Friedrich Miescher Institute for Biomedical Research Lats et cancer du sein
WO2016089883A1 (fr) 2014-12-01 2016-06-09 Novartis Ag Compositions et méthodes pour le diagnostic et le traitement du cancer de la prostate
EP3302525A2 (fr) 2015-06-05 2018-04-11 Novartis AG Méthodes et compositions permettant de diagnostiquer, traiter et surveiller le traitement de troubles associés à une déficience en shank3
US20180348224A1 (en) 2015-10-28 2018-12-06 Friedrich Miescher Institute For Biomedical Resear Ch Tenascin-w and biliary tract cancers
PH12018501207B1 (en) 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
US9721645B1 (en) 2016-01-29 2017-08-01 Taiwan Semiconductor Manufacturing Company, Ltd. SRAM arrays and methods of manufacturing same
WO2018047148A1 (fr) 2016-09-12 2018-03-15 Novartis Ag Composés pour inhibition du miarn
EP3535396A1 (fr) 2016-11-01 2019-09-11 Novartis AG Procédés et compositions pour améliorer l'édition de gènes
US11530413B2 (en) 2017-07-21 2022-12-20 Novartis Ag Compositions and methods to treat cancer
KR20240161202A (ko) 2017-09-11 2024-11-12 애로우헤드 파마슈티컬스 인코포레이티드 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물
WO2019213276A1 (fr) 2018-05-02 2019-11-07 Novartis Ag Régulateurs de cellules souches pluripotentes humaines et leurs utilisations
SG11202105577TA (en) 2019-01-09 2021-06-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
TW202440941A (zh) 2022-12-20 2024-10-16 瑞士商諾華公司 核酸連接方法
TW202440911A (zh) 2022-12-20 2024-10-16 瑞士商諾華公司 工程化連接酶及其用途
WO2025262452A1 (fr) 2024-06-18 2025-12-26 Novartis Ag Procédé de ligature d'acide nucléique

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69034150T2 (de) * 1989-10-24 2005-08-25 Isis Pharmaceuticals, Inc., Carlsbad 2'-Modifizierte Oligonukleotide
US5670633A (en) * 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US6005087A (en) * 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6399754B1 (en) * 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
KR930016437A (ko) 1992-01-22 1993-08-26 귀틀라인, 슈미트 올리고뉴클레오티드 유사체, 이의 제조방법 및 용도
US6033909A (en) * 1992-01-22 2000-03-07 Hoechst Aktiengesellschaft Oligonucleotide analogs, their preparation and use
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5976567A (en) * 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
WO2002096927A2 (fr) * 2001-05-29 2002-12-05 Ribozyme Pharmaceuticals, Incorporated Modulation a base de l'acide nucleique des maladies et troubles de l'appareil reproducteur chez la femme
US5998203A (en) * 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
AU733310C (en) * 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
RU2217161C2 (ru) * 1997-08-26 2003-11-27 Сосьете Де Консей Де Решерш Э Д'Аппликасьон Сьентифик (С.К.Р.А.С.) Способ лечения вирусного гепатита
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6211349B1 (en) * 1998-12-30 2001-04-03 Oligos Etc., Inc. Protonated/acidified nucleic acids and methods of use
CA2361201A1 (fr) * 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition et methode destinees a l'attenuation in vivo et in vitro de l'expression genique utilisant de l'arn double brin
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US20050233329A1 (en) * 2002-02-20 2005-10-20 Sirna Therapeutics, Inc. Inhibition of gene expression using duplex forming oligonucleotides
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
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)
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
CA2429814C (fr) * 2000-12-01 2014-02-18 Thomas Tuschl Petites molecules d'arn mediant l'interference arn
CA2369944A1 (fr) 2001-01-31 2002-07-31 Nucleonics Inc. Utilisation de l'inhibition genetique post-transcriptionnelle pour identifier les sequences d'acides nucleiques qui modulent la fonction d'une cellule
EP1386004A4 (fr) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc Modulation de l'expression genique associee a la proliferation inflammatoire et a la croissance de neurites, par des procedes faisant intervenir l'acide nucleique
US20030175950A1 (en) * 2001-05-29 2003-09-18 Mcswiggen James A. RNA interference mediated inhibition of HIV gene expression using short interfering RNA
US20070032441A1 (en) * 2001-05-18 2007-02-08 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
US20040019001A1 (en) * 2002-02-20 2004-01-29 Mcswiggen James A. RNA interference mediated inhibition of protein typrosine phosphatase-1B (PTP-1B) gene expression using short interfering RNA
AU2004266311B2 (en) 2001-05-18 2009-07-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20090247606A1 (en) * 2001-08-28 2009-10-01 Sirna Therapeutics, Inc. RNA Interference Mediated Inhibition of Adenosine A1 Receptor (ADORA1) Gene Expression Using Short Interfering Nucleic Acid (siNA)
US8258288B2 (en) * 2002-02-20 2012-09-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
EP1432724A4 (fr) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Inhibition a mediation par interference d'arn de genes de map kinase
DK1389637T3 (da) * 2002-08-05 2012-09-03 Silence Therapeutics Ag Interfererende RNA-molekyler med stumpe ender
DE60310944T3 (de) * 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
JP4605799B2 (ja) * 2003-04-02 2011-01-05 ダーマコン， インコーポレイテッド Ｒｎａ干渉において使用するための修飾ポリヌクレオチド
WO2004092383A2 (fr) * 2003-04-15 2004-10-28 Sirna Therapeutics, Inc. Inhibition par interference d'arn de l'expression genetique virale du syndrome respiratoire aigu severe au moyen d'acide nucleique d'interference court
ES2702942T3 (es) * 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
WO2005009346A2 (fr) * 2003-06-24 2005-02-03 Mirus Corporation Inhibition d'une fonction genique par distribution in vivo d'inhibiteurs d'expression genique a base de polynucleotides dans des cellules mammiferes
EP1660657A1 (fr) 2003-08-28 2006-05-31 Novartis AG Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'
AU2005247509C1 (en) 2004-05-27 2012-09-20 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds

Also Published As

Publication number Publication date
CN1845993B (zh) 2010-06-23
BRPI0413146A (pt) 2006-10-03
CN1845993A (zh) 2006-10-11
RU2010119292A (ru) 2011-12-10
EP2489737A1 (fr) 2012-08-22
IS8358A (is) 2006-03-17
EP2338995A2 (fr) 2011-06-29
JP2007503803A (ja) 2007-03-01
KR20060087531A (ko) 2006-08-02
EP1660657A1 (fr) 2006-05-31
US20070203084A1 (en) 2007-08-30
RU2399671C2 (ru) 2010-09-20
US8097716B2 (en) 2012-01-17
SG152279A1 (en) 2009-05-29
AU2004269150C1 (en) 2010-11-18
CO5680494A2 (es) 2006-09-29
JP2011160796A (ja) 2011-08-25
US20090192113A1 (en) 2009-07-30
CA2536333C (fr) 2013-01-08
IL173565A (en) 2012-07-31
IL173565A0 (en) 2006-07-05
AU2004269150A1 (en) 2005-03-10
CA2536333A1 (fr) 2005-03-10
TNSN06065A1 (en) 2007-10-03
RU2006109547A (ru) 2007-10-10
CN101914533A (zh) 2010-12-15
AU2004269150B2 (en) 2009-06-04
ZA200601003B (en) 2007-04-25
NO20061345L (no) 2006-05-29
WO2005021749A1 (fr) 2005-03-10
KR20080034046A (ko) 2008-04-17
KR20110007263A (ko) 2011-01-21
CN101914533B (zh) 2013-06-19
ECSP066388A (es) 2006-08-30
EP2338995A3 (fr) 2012-01-11
MXPA06002216A (es) 2006-04-27
KR101059315B1 (ko) 2011-08-24
NZ545360A (en) 2009-06-26

Similar Documents

Publication Publication Date Title
MA28029A1 (fr) Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'
EP1010691A3 (fr) Dérives de l'hydrazide, leur procédé de préparation et les compositions pharmaceutiques qui les contiennent
ATE239752T1 (de) Pyrimidinderivate als markierte bindungspartner
ATE277066T1 (de) Bicyclonucleosid-analoga
CA2192824A1 (fr) Derives de l'acide pyridone-carboxylique, procedes de preparation connexe et agents anti-tumorals contenant ces derives
WO1999035157B1 (fr) Nouveaux derives d'erythromycine
EP1285908A4 (fr) Derives substitues d'acide phenylpropionique
KR910002873A (ko) 티아졸로[5,4-b] 아제핀 화합물
FR2786188B1 (fr) Nouveaux derives de l'erythromycine, leur procede de preparation et leur applicaion comme medicaments
CA1106277A (fr) Agents microbicide a base de sels d'alkyldiguanidinium
EA200200523A1 (ru) Новые разветвленные замещенные аминопроизводные 3-амино-1-фенил-1h[1,2,4]триазола, способы их получения и содержащие их фармацевтические композиции
DK0455300T3 (da) Barbitursyrederivater med insekticid virkning
PT1016409E (pt) Composicao liofilizada esfingoglicolipidos e o processo para a sua preparacao
KR900701779A (ko) 옥시란유도체 및 그것을 유효성분으로 하는 제초제
KR870007901A (ko) N-치환된 사프라마이신 유도체 및 이의
CA2118052A1 (fr) Derives du n-(aminosubstitue)pyrrole, leur procede de preparation et compositions herbicides
RU2008112549A (ru) Сочетание биологически доступного метионина с по меньшей мере одним эфирным маслом
BR9908960A (pt) Amino guanidinas cìclicas
EP1252878A3 (fr) Agent pour la croissance des cheveux
CA2526052A1 (fr) Derives d'indazol utilises pour traiter les douleurs neuropathiques
KR970019858A (ko) 이미노아세테이트를 포함하는 신규 티아졸 화합물, 그의 제조방법 및 유효성분으로서 그를 함유하는 살균제 조성물
EP0246171A3 (fr) Compositions fongicides à base de dérivés nicotiniques, nouveaux dérivés nicotiniques et leur préparation
GB2250989B (en) Thioacid amide derivatives and fodder compositions containing the same
CA2212449A1 (fr) Preparation amelioree de 1,2,5,6-tetrahydro-3-carboalcoxypyridines comme l'arecoline et de sels de 1,2,5,6-tetrahydro-3-carboalcoxypyridines et d'hydrobromure d'arecoline
EP0273732A3 (fr) Dérivés de streptovaricine C comme remède contre le virus du SIDA