JP4605799B2 - Ｒｎａ干渉において使用するための修飾ポリヌクレオチド - Google Patents

Ｒｎａ干渉において使用するための修飾ポリヌクレオチド Download PDF

Info

Publication number
JP4605799B2
JP4605799B2 JP2006509678A JP2006509678A JP4605799B2 JP 4605799 B2 JP4605799 B2 JP 4605799B2 JP 2006509678 A JP2006509678 A JP 2006509678A JP 2006509678 A JP2006509678 A JP 2006509678A JP 4605799 B2 JP4605799 B2 JP 4605799B2
Authority
JP
Japan
Prior art keywords
sirna
modification
sense
nucleotide
antisense
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
JP2006509678A
Other languages
English (en)
Japanese (ja)
Other versions
JP2007525169A (ja
JP2007525169A5 (https=
Inventor
デビン リーク，
アンジェラ レイノルズ，
アンジェラ フボロバ，
ウィリアム マーシャル，
ユリー フェデロブ，
キンバーリー ニコルス，
Original Assignee
ダーマコン， インコーポレイテッド
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
Priority claimed from US10/406,908 external-priority patent/US20040198640A1/en
Application filed by ダーマコン， インコーポレイテッド filed Critical ダーマコン， インコーポレイテッド
Publication of JP2007525169A publication Critical patent/JP2007525169A/ja
Publication of JP2007525169A5 publication Critical patent/JP2007525169A5/ja
Application granted granted Critical
Publication of JP4605799B2 publication Critical patent/JP4605799B2/ja
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Images

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N2310/32—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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Microbiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2006509678A 2003-04-02 2004-04-01 Ｒｎａ干渉において使用するための修飾ポリヌクレオチド Expired - Lifetime JP4605799B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US10/406,908 US20040198640A1 (en) 2003-04-02 2003-04-02 Stabilized polynucleotides for use in RNA interference
US10/613,077 US20040266707A1 (en) 2003-04-02 2003-07-01 Stabilized polynucleotides for use in RNA interference
US54264604P 2004-02-06 2004-02-06
US54266804P 2004-02-06 2004-02-06
US54366104P 2004-02-10 2004-02-10
US54364004P 2004-02-10 2004-02-10
PCT/US2004/010343 WO2004090105A2 (en) 2003-04-02 2004-04-01 Modified polynucleotides for use in rna interference

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2010106021A Division JP5468978B2 (ja) 2003-04-02 2010-04-30 Ｒｎａ干渉において使用するための修飾ポリヌクレオチド

Publications (3)

Publication Number Publication Date
JP2007525169A JP2007525169A (ja) 2007-09-06
JP2007525169A5 JP2007525169A5 (https=) 2010-06-24
JP4605799B2 true JP4605799B2 (ja) 2011-01-05

Family

ID=33163279

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2006509678A Expired - Lifetime JP4605799B2 (ja) 2003-04-02 2004-04-01 Ｒｎａ干渉において使用するための修飾ポリヌクレオチド

Country Status (3)

Country Link
EP (1) EP1608733B1 (https=)
JP (1) JP4605799B2 (https=)
WO (1) WO2004090105A2 (https=)

Families Citing this family (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US7517864B2 (en) 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
ATE556714T1 (de) 2002-02-01 2012-05-15 Life Technologies Corp Doppelsträngige oligonukleotide
GB2413557B (en) * 2002-02-20 2006-08-16 Sirna Therapeutics Inc RNA interference mediated inhibtion of gene expression using chemically modified 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)
US7612196B2 (en) 2002-11-14 2009-11-03 Dharmacon, Inc. siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B)
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7619081B2 (en) 2002-11-14 2009-11-17 Dharmacon, Inc. siRNA targeting coatomer protein complex, subunit beta 2 (COPB2)
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7592442B2 (en) 2002-11-14 2009-09-22 Dharmacon, Inc. siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2)
US7691998B2 (en) 2002-11-14 2010-04-06 Dharmacon, Inc. siRNA targeting nucleoporin 62kDa (Nup62)
US9719092B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting CNTD2
US9839649B2 (en) 2002-11-14 2017-12-12 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7635770B2 (en) 2002-11-14 2009-12-22 Dharmacon, Inc. siRNA targeting protein kinase N-3 (PKN-3)
US9771586B2 (en) 2002-11-14 2017-09-26 Thermo Fisher Scientific Inc. RNAi targeting ZNF205
US10011836B2 (en) 2002-11-14 2018-07-03 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7951935B2 (en) 2002-11-14 2011-05-31 Dharmacon, Inc. siRNA targeting v-myc myelocytomatosis viral oncogene homolog (MYC)
US9719094B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting SEC61G
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7977471B2 (en) 2002-11-14 2011-07-12 Dharmacon, Inc. siRNA targeting TNFα
US8198427B1 (en) 2002-11-14 2012-06-12 Dharmacon, Inc. SiRNA targeting catenin, beta-1 (CTNNB1)
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US20040167090A1 (en) * 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
EP1660657A1 (en) 2003-08-28 2006-05-31 Novartis AG Interfering rna duplex having blunt-ends and 3'-modifications
US20050176045A1 (en) * 2004-02-06 2005-08-11 Dharmacon, Inc. SNP discriminatory siRNA
WO2005078094A2 (en) * 2004-02-06 2005-08-25 Dharmacon, Inc. Stabilized rnas as transfection controls and silencing reagents
ATE452188T1 (de) 2004-02-10 2010-01-15 Sirna Therapeutics Inc Rna-interferenz-vermittelte hemmung der genexpression unter verwendung multifunktioneller sina (short interfering nucleic acid)
KR101147147B1 (ko) * 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US7605250B2 (en) 2004-05-12 2009-10-20 Dharmacon, Inc. siRNA targeting cAMP-specific phosphodiesterase 4D
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
EP2302055B1 (en) * 2004-11-12 2014-08-27 Asuragen, Inc. Methods and compositions involving miRNA and miRNA inhibitor molecules
EP1814994A4 (en) * 2004-11-22 2010-07-14 Dharmacon Inc APPARATUS AND SYSTEM COMPRISING DRY EXTINCTION DRY COMPOSITIONS
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
CN101346393B (zh) * 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
AU2007299705B2 (en) 2006-09-22 2012-09-06 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
KR101629017B1 (ko) 2007-05-22 2016-06-10 아크투루스 쎄라퓨틱스, 인크. 히드록시메틸 치환된 ｒｎａ 올리고뉴클레오티드 및 ｒｎａ 복합체
CL2008002775A1 (es) 2007-09-17 2008-11-07 Amgen Inc Uso de un agente de unión a esclerostina para inhibir la resorción ósea.
US8318496B2 (en) 2007-10-04 2012-11-27 Isis Pharmaceuticals, Inc. Compounds and methods for improving cellular uptake of oligomeric compounds
CN101896201A (zh) 2007-12-14 2010-11-24 安进公司 使用抗硬骨素抗体治疗骨折的方法
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
EP2264167B1 (en) * 2008-03-31 2016-10-12 National Institute of Advanced Industrial Science and Technology Double-stranded lipid-modified rna having high rna interference effect
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
SG171879A1 (en) 2008-12-03 2011-07-28 Marina Biotech Inc Usirna complexes
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2454371B1 (en) 2009-07-13 2021-01-20 Somagenics, Inc. Chemical modification of small hairpin rnas for inhibition of gene expression
WO2011072082A2 (en) * 2009-12-09 2011-06-16 Nitto Denko Corporation Modulation of hsp47 expression
US8877439B2 (en) 2009-12-17 2014-11-04 Sirna Therapeutics, Inc. Method for rapidly evaluating performance of short interfering RNA with novel chemical modifications
EP3560503B1 (en) 2010-03-24 2021-11-17 Phio Pharmaceuticals Corp. Rna interference in dermal and fibrotic indications
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
WO2011125943A1 (ja) * 2010-04-01 2011-10-13 日本新薬株式会社 修飾オリゴヌクレオチド
MX2013000986A (es) * 2010-07-28 2013-07-03 Alcon Res Ltd Sirna dirigido al vegfa y metodos de tratamiento in vivo.
US9243246B2 (en) 2010-08-24 2016-01-26 Sirna Therapeutics, Inc. Single-stranded RNAi agents containing an internal, non-nucleic acid spacer
JP2013541510A (ja) * 2010-08-31 2013-11-14 メルク・シャープ・エンド・ドーム・コーポレイション 新規の単一の化学物質、およびオリゴヌクレオチドの送達方法
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)
CN106061488B (zh) 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
US9856475B2 (en) 2014-03-25 2018-01-02 Arcturus Therapeutics, Inc. Formulations for treating amyloidosis
JP6771387B2 (ja) 2014-03-25 2020-10-21 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的ｕｎａオリゴマー
AU2015236215B2 (en) 2014-03-25 2020-03-19 Arcturus Therapeutics, Inc. UNA oligomers having reduced off-target effects in gene silencing
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
US10900039B2 (en) 2014-09-05 2021-01-26 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
US10519447B2 (en) 2015-04-01 2019-12-31 Arcturus Therapeutics, Inc. Therapeutic UNA oligomers and uses thereof
EP3862005A1 (en) 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
WO2017015671A1 (en) 2015-07-23 2017-01-26 Arcturus Therapeutics, Inc. Compositions for treating amyloidosis
US10548990B2 (en) 2015-07-31 2020-02-04 Genecare Research Institute Co., Ltd. Modified siRNA and pharmaceutical composition
WO2017053681A1 (en) 2015-09-24 2017-03-30 Wisconsin Alumni Research Foundation Methods of expanding hematopoietic stem cells, compositions, and methods of use thereof
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
WO2017079227A1 (en) 2015-11-05 2017-05-11 University Of Connecticut Compositions and methods for the treatment of liver fibrosis
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
US12544344B2 (en) 2017-04-19 2026-02-10 Phio Pharmaceuticals Corp. Topical delivery of nucleic acid compounds
CN111527206A (zh) * 2017-11-13 2020-08-11 赛伦斯治疗有限责任公司 用于抑制靶标基因表达的包含二硫代磷酸酯键的核酸
AU2018378812B2 (en) 2017-12-06 2025-05-22 Avidity Biosciences, Inc. Compositions and methods of treating muscle atrophy and myotonic dystrophy
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2022212208A1 (en) * 2021-03-29 2022-10-06 University Of Massachusetts Oligonucleotides for syngr-3 modulation
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
US20250340883A1 (en) * 2021-11-18 2025-11-06 Resonance Health Analysis Services Pty Ltd Method For Treating Cyclophilin B Associated Diseases

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
CA2429814C (en) * 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
WO2003012052A2 (en) * 2001-07-30 2003-02-13 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Specific inhibition of gene expression by small double stranded rnas

Also Published As

Publication number Publication date
WO2004090105A2 (en) 2004-10-21
EP1608733A2 (en) 2005-12-28
EP1608733B1 (en) 2011-12-07
JP2007525169A (ja) 2007-09-06
EP1608733A4 (en) 2008-02-27
WO2004090105A3 (en) 2006-07-13

Similar Documents

Publication Publication Date Title
JP4605799B2 (ja) Ｒｎａ干渉において使用するための修飾ポリヌクレオチド
JP2007525169A5 (https=)
EP2261334B1 (en) Modified polynucleotides for use in RNA interference
EP2123759B1 (en) Stabilized RNAS as transfection controls and silencing reagents
JP5112854B2 (ja) Ｒｎａ干渉においてオフターゲット効果を減じるための修飾されたポリヌクレオチド類
Chi et al. Safety of antisense oligonucleotide and siRNA-based therapeutics
JP5816556B2 (ja) 治療剤のためのｕｎａオリゴマー構造
JP2025072451A (ja) 安定性が増加した修飾オリゴヌクレオチド
EP1986697B1 (en) Compositions and methods for inhibiting gene silencing by rna interference
US8604183B2 (en) Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US20040058886A1 (en) Short interfering RNAs having a hairpin structure containing a non-nucleotide loop
US20060223777A1 (en) Highly functional short hairpin RNA
WO2005078095A1 (en) SNP DISCRIMINATORY siRNA
EP1768998A2 (en) Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
CN121294438A (zh) 高级rna靶向(arnatar)
US20090280567A1 (en) Stabilized sirnas as transfection controls and silencing reagents
JP5468978B2 (ja) Ｒｎａ干渉において使用するための修飾ポリヌクレオチド

Legal Events

Date Code Title Description
A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20100202

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20100430

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20100623

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20100907

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20100929

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20101004

R150 Certificate of patent or registration of utility model

Ref document number: 4605799

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

Free format text: JAPANESE INTERMEDIATE CODE: R150

FPAY Renewal fee payment (event date is renewal date of database)

Free format text: PAYMENT UNTIL: 20131015

Year of fee payment: 3

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

S533 Written request for registration of change of name

Free format text: JAPANESE INTERMEDIATE CODE: R313533

R350 Written notification of registration of transfer

Free format text: JAPANESE INTERMEDIATE CODE: R350

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

S533 Written request for registration of change of name

Free format text: JAPANESE INTERMEDIATE CODE: R313533

R350 Written notification of registration of transfer

Free format text: JAPANESE INTERMEDIATE CODE: R350

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

EXPY Cancellation because of completion of term