JP6106085B2 - 内部非核酸スペーサーを含む一本鎖RNAi剤 - Google Patents

内部非核酸スペーサーを含む一本鎖RNAi剤 Download PDF

Info

Publication number
JP6106085B2
JP6106085B2 JP2013526032A JP2013526032A JP6106085B2 JP 6106085 B2 JP6106085 B2 JP 6106085B2 JP 2013526032 A JP2013526032 A JP 2013526032A JP 2013526032 A JP2013526032 A JP 2013526032A JP 6106085 B2 JP6106085 B2 JP 6106085B2
Authority
JP
Japan
Prior art keywords
nucleotide
molecule
nucleotides
nucleic acid
spacer
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.)
Active
Application number
JP2013526032A
Other languages
English (en)
Japanese (ja)
Other versions
JP2013535982A (ja
Inventor
コーン,ギヨーム
リム,リー
ウイリンガム,アーロン
チヨー,リーホン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirna Therapeutics Inc
Original Assignee
Sirna Therapeutics Inc
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 Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Publication of JP2013535982A publication Critical patent/JP2013535982A/ja
Application granted granted Critical
Publication of JP6106085B2 publication Critical patent/JP6106085B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183Diol linkers, e.g. glycols or propanediols
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2013526032A 2010-08-24 2011-08-19 内部非核酸スペーサーを含む一本鎖RNAi剤 Active JP6106085B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US37647110P 2010-08-24 2010-08-24
US61/376,471 2010-08-24
PCT/US2011/048338 WO2012027206A1 (en) 2010-08-24 2011-08-19 SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2016249699A Division JP2017079776A (ja) 2010-08-24 2016-12-22 内部非核酸スペーサーを含む一本鎖RNAi剤

Publications (2)

Publication Number Publication Date
JP2013535982A JP2013535982A (ja) 2013-09-19
JP6106085B2 true JP6106085B2 (ja) 2017-03-29

Family

ID=45723747

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2013526032A Active JP6106085B2 (ja) 2010-08-24 2011-08-19 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2016249699A Pending JP2017079776A (ja) 2010-08-24 2016-12-22 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2020009687A Withdrawn JP2020074788A (ja) 2010-08-24 2020-01-24 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2021199768A Pending JP2022037070A (ja) 2010-08-24 2021-12-09 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2023076252A Active JP7527437B2 (ja) 2010-08-24 2023-05-02 内部非核酸スペーサーを含む一本鎖RNAi剤

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2016249699A Pending JP2017079776A (ja) 2010-08-24 2016-12-22 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2020009687A Withdrawn JP2020074788A (ja) 2010-08-24 2020-01-24 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2021199768A Pending JP2022037070A (ja) 2010-08-24 2021-12-09 内部非核酸スペーサーを含む一本鎖RNAi剤
JP2023076252A Active JP7527437B2 (ja) 2010-08-24 2023-05-02 内部非核酸スペーサーを含む一本鎖RNAi剤

Country Status (6)

Country Link
US (3) US9243246B2 (OSRAM)
EP (2) EP2609198B8 (OSRAM)
JP (5) JP6106085B2 (OSRAM)
KR (1) KR20130137160A (OSRAM)
CN (1) CN103140582A (OSRAM)
WO (1) WO2012027206A1 (OSRAM)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
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)
US8748405B2 (en) * 2007-01-26 2014-06-10 City Of Hope Methods and compositions for the treatment of cancer or other diseases
WO2011028550A1 (en) 2009-08-24 2011-03-10 Merck Sharp & Dohme Corp. Segmented micro rna mimetics
EP2609198B8 (en) * 2010-08-24 2018-03-28 Sirna Therapeutics, Inc. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
EP3327125B1 (en) 2010-10-29 2020-08-05 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
WO2012083005A2 (en) 2010-12-15 2012-06-21 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
KR20140091688A (ko) 2011-10-06 2014-07-22 미라젠 세러퓨틱스 인코포레이티드 마이크로rna 조절에 의한 전신 에너지 항상성의 제어
EP3358013B1 (en) 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
WO2013170146A1 (en) 2012-05-10 2013-11-14 Uab Research Foundation Methods and compositions for modulating mir-204 activity
US20140378526A1 (en) * 2012-05-11 2014-12-25 City Of Hope Design of nucleic acid binding molecules with non-watson crick and non-canonical pairing based on artificial mutation consensus sequences to counter escape mutations
EP3747891A3 (en) * 2012-05-21 2021-03-24 Agilent Technologies, Inc. Compositions and methods for conjugating oligonucleotides
EP3536789A1 (en) 2012-06-06 2019-09-11 Boehringer Ingelheim International GmbH Cell engineering using rnas
CA2876180C (en) 2012-06-21 2019-11-19 MiRagen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
EP3052107B1 (en) * 2013-10-04 2018-05-02 Novartis AG Organic compounds to treat hepatitis b virus
EP3722277A3 (en) 2013-10-04 2021-01-20 Novartis AG 3'end caps for rna-interferring agents for use in rna
CN104560992B (zh) * 2013-10-10 2018-01-02 复旦大学附属妇产科医院 一种用于预防和治疗人乳头瘤病毒感染和宫颈癌的微小rna
KR101696704B1 (ko) * 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
WO2015106255A1 (en) 2014-01-13 2015-07-16 City Of Hope Multivalent oligonucleotide assemblies
CN103924002B (zh) * 2014-05-04 2016-01-20 山东大学 血清中的microRNA作为肝癌诊断标志物的应用
JP6824736B2 (ja) 2014-06-12 2021-02-03 東レ株式会社 前立腺がんの検出キット又はデバイス及び検出方法
SG11201705907XA (en) 2015-01-20 2017-08-30 Miragen Therapeutics Inc Mir-92 inhibitors and uses thereof
AU2016308694A1 (en) 2015-08-20 2018-02-22 Asociación Centro De Investigación Cooperativa En Biociencias-Cic Biogune Methods and compositions to treat liver diseases and conditions
TWI685500B (zh) * 2015-10-13 2020-02-21 長庚大學 以微核酸-520b(miR-520b)序列作爲抑制頭頸癌腫瘤生長、侵犯與轉移及其醫藥組成物之用途
EP3386517A4 (en) * 2015-12-08 2019-08-07 Solstice Biologics, Ltd. POLYNUCLEOTIDE CONSTRUCTS COMPRISING AN AUXILIARY FRACTION RELATED NON-BIOREVERSIBLE TO AN INTERNETOSIDIC PHOSPHATE OR PHOSPHOROTHIOATE
WO2017181088A1 (en) * 2016-04-14 2017-10-19 University Of Florida Research Foundation, Incorporated Use of mir-223-3p as a cancer therapeutic and method for treating cancer using the same
CN105861728B (zh) * 2016-06-12 2024-11-19 上海市第十人民医院 循环miRNA作为年龄相关黄斑变性诊断标志物中的应用
CN107630015B (zh) * 2016-07-19 2021-03-09 上海市东方医院 一种稳定的dna-rna双链结构
WO2018035380A1 (en) 2016-08-17 2018-02-22 Solstice Biologics, Ltd. Polynucleotide constructs
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
JP7162918B2 (ja) * 2018-05-31 2022-10-31 コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション マイクロrnaを抑制する変形核酸及びその用途
WO2020032228A1 (ja) 2018-08-10 2020-02-13 東レ株式会社 前立腺がんの検出のためのキット、デバイス及び方法
CN109706239A (zh) * 2018-09-26 2019-05-03 南京市妇幼保健院 一种miR-1976在防治细菌性阴道病中的应用
US20240191229A1 (en) * 2021-04-13 2024-06-13 Adarx Pharmaceuticals, Inc. Rna silencing agents and methods of use
IL315546A (en) 2022-03-16 2024-11-01 Empirico Inc GALNAC compositions to improve siRNA bioavailability
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
CN114767703B (zh) * 2022-06-20 2022-09-02 山东恺悌生物制品有限公司 miR-4311模拟物在制备肺癌治疗药物中的应用
WO2024249791A2 (en) * 2023-06-02 2024-12-05 Quralis Corporation Modified unc13a oligonucleotides
US12491257B2 (en) * 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates

Family Cites Families (201)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5495009A (en) 1989-10-24 1996-02-27 Gilead Sciences, Inc. Oligonucleotide analogs containing thioformacetal linkages
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
US5962219A (en) 1990-06-11 1999-10-05 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: chemi-selex
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
PL169576B1 (pl) 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
AU4770093A (en) 1992-07-02 1994-01-31 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
CA2140343A1 (en) 1992-07-17 1994-02-03 Sean M. Sullivan Method and reagent for treatment of animal diseases
US6174868B1 (en) 1992-09-10 2001-01-16 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis C virus-associated diseases
US6235886B1 (en) 1993-09-03 2001-05-22 Isis Pharmaceuticals, Inc. Methods of synthesis and use
CA2154363A1 (en) 1993-01-22 1994-08-04 Bruce A. Sullenger Localization of therapeutic agents
US5767264A (en) 1993-01-22 1998-06-16 Mta Zozponti Kemiai Kutato Intezet Oligodeoxynucleotides containing 5-alkyl, 5-(1-alkenyl)- and 5-(1-alkynl) pyrimidines
US6410322B1 (en) 1993-07-27 2002-06-25 Hybridon Inc Antisense oligonucleotide inhibition of vascular endothelial growth factor expression
DE69431669T2 (de) 1993-09-02 2003-10-23 Ribozyme Pharmaceuticals, Inc. Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
DE69433036T2 (de) 1993-09-03 2004-05-27 Isis Pharmaceuticals, Inc., Carlsbad Aminoderivatisierte nukleoside und oligonukleoside
US5624803A (en) 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US5861288A (en) 1993-10-18 1999-01-19 Ribozyme Pharmaceuticals, Inc. Catalytic DNA
DE69415343T2 (de) 1993-10-27 1999-08-26 Ribozyme Pharmaceuticals 2'-amido-und 2'-peptido-modifizierte oligonukleotide
DE4338704A1 (de) 1993-11-12 1995-05-18 Hoechst Ag Stabilisierte Oligonucleotide und deren Verwendung
US5587471A (en) 1994-01-11 1996-12-24 Isis Pharmaceuticals, Inc. Method of making oligonucleotide libraries
US5902880A (en) 1994-08-19 1999-05-11 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US6447796B1 (en) 1994-05-16 2002-09-10 The United States Of America As Represented By The Secretary Of The Army Sustained release hydrophobic bioactive PLGA microspheres
US6146886A (en) 1994-08-19 2000-11-14 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
CA2207593A1 (en) 1994-12-13 1996-06-20 John Gustofson Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5968909A (en) 1995-08-04 1999-10-19 Hybridon, Inc. Method of modulating gene expression with reduced immunostimulatory response
JP2000501414A (ja) 1995-11-22 2000-02-08 ザ・ジョンズ・ホプキンス・ユニバーシティー 生体分子の細胞取り込みを高めるリガンド
AU1430097A (en) 1996-01-16 1997-08-11 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
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
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
AU5720498A (en) 1996-12-24 1998-07-17 Ribozyme Pharmaceuticals, Inc. Chemical synthesis of nucleosides analogs and their incorporation into polynucleotides
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US6235310B1 (en) 1997-04-04 2001-05-22 Valentis, Inc. Methods of delivery using cationic lipids and helper lipids
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
TWI225412B (en) 1997-06-23 2004-12-21 Sequus Pharm Inc Liposome-entrapped polynucleotide composition and method
US6395713B1 (en) 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
AR013269A1 (es) 1997-08-04 2000-12-13 Scras Producto que contiene por lo menos un rna de doble filamento combinado con por lo menos un agente anti-viral, para la utilizacion terapeutica en eltratamiento de una enfermedad viral, en especial de la hepatitis viral
WO1999014226A2 (en) 1997-09-12 1999-03-25 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
WO1999031262A2 (en) 1997-12-16 1999-06-24 Valentis, Inc. Needle-free injection of formulated nucleic acid molecules
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
EP2138191A1 (en) 1998-01-05 2009-12-30 University Of Washington Enhanced transport using membrane disruptive agents
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
EP1071753A2 (en) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
CA2330574A1 (en) 1998-04-29 1999-11-04 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
GB9827152D0 (en) 1998-07-03 1999-02-03 Devgen Nv Characterisation of gene function using double stranded rna inhibition
EP1096921B1 (en) 1998-07-20 2003-04-16 Protiva Biotherapeutics Inc. Liposomal encapsulated nucleic acid-complexes
WO2000025248A2 (en) 1998-10-26 2000-05-04 Intervu, Inc. Audience management for interactive network events
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
ATE408699T1 (de) 1999-03-10 2008-10-15 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
KR20020013513A (ko) 1999-03-18 2002-02-20 추후제출 Xylo-lna 유사체
ATE332909T1 (de) 1999-03-24 2006-08-15 Exiqon As Verbesserte synthese für 2.2.1.öbicyclo- nukleoside
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
KR20070086708A (ko) 1999-07-14 2007-08-27 알자 코포레이션 중성 지질중합체 및 그를 함유하는 리포솜 조성물
EP1212416A2 (en) 1999-08-31 2002-06-12 Ribozyme Pharmaceuticals, Inc. Nucleic acid based modulators of gene expression
WO2001025248A2 (en) 1999-10-04 2001-04-12 Exiqon A/S Design of high affinity rnase h recruiting oligonucleotide
HK1047109A1 (zh) 1999-10-15 2003-02-07 University Of Massachusetts 作为指定基因干预工具的rna干预轨迹基因
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US6602857B1 (en) 2000-01-18 2003-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US6401406B1 (en) 2000-02-11 2002-06-11 Domald K. Komara Retainment device for concrete block inspection plates
WO2005041859A2 (en) 2003-04-30 2005-05-12 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery.
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
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
WO2005019453A2 (en) 2001-05-18 2005-03-03 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
US20030190635A1 (en) 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US6613567B1 (en) 2000-09-15 2003-09-02 Isis Pharmaceuticals, Inc. Antisense inhibition of Her-2 expression
JP4413493B2 (ja) 2000-10-04 2010-02-10 サンタリス ファーマ アー/エス プリンlna類似体の改善された合成方法
US7427394B2 (en) 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
US6906182B2 (en) 2000-12-01 2005-06-14 Cell Works Therapeutics, Inc. Conjugates of glycosylated/galactosylated peptide, bifunctional linker, and nucleotidic monomers/polymers, and related compositions and method of use
US20020130430A1 (en) 2000-12-29 2002-09-19 Castor Trevor Percival Methods for making polymer microspheres/nanospheres and encapsulating therapeutic proteins and other products
US20050164220A1 (en) 2001-03-19 2005-07-28 Decode Genetics Ehf. Susceptibility gene for human stroke: method of treatment
WO2004028341A2 (en) 2001-03-19 2004-04-08 Decode Genetics Ehf. Susceptibility gene for human stroke; methods of treatment
JP2004532022A (ja) 2001-03-26 2004-10-21 サーナ・セラピューティクス・インコーポレイテッド B型肝炎ウイルスおよびc型肝炎ウイルスの複製のオリゴヌクレオチド媒介性阻害
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
WO2003070917A2 (en) 2002-02-20 2003-08-28 Sirna Therapeutics, Inc. Rna interference mediated inhibition of myc and myb genes or genes of their respective pathways
US20050148530A1 (en) 2002-02-20 2005-07-07 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)
AU2002317437A1 (en) 2001-05-18 2002-12-03 Cureon A/S Therapeutic uses of lna-modified oligonucleotides in infectious diseases
WO2003070884A2 (en) 2002-02-20 2003-08-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF MDR P-GLYCOPROTEIN 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
WO2003072590A1 (en) 2002-02-20 2003-09-04 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
WO2003070197A2 (en) 2002-02-20 2003-08-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF TGF-BETA AND TGF-BETA RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
WO2003070743A1 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF G72 AND D-AMINO ACID OXIDASE (DAAO) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2003070912A2 (en) 2001-06-06 2003-08-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF EPIDERMAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING 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
WO2003070910A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20030175950A1 (en) 2001-05-29 2003-09-18 Mcswiggen James A. RNA interference mediated inhibition of HIV gene expression using short interfering RNA
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)
WO2004097020A2 (en) 2003-04-25 2004-11-11 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase gene expression
US20050196781A1 (en) 2001-05-18 2005-09-08 Sirna Therapeutics, Inc. RNA interference mediated inhibition of STAT3 gene expression using short interfering nucleic acid (siNA)
EP3231445A1 (en) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
CA2448320A1 (en) 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Ribozyme based treatment of female reproductive diseases
AU2002328792A1 (en) 2001-07-12 2003-01-29 Santaris Pharma A/S Method for preparation of lna phosphoramidites
ATE481497T1 (de) 2001-08-01 2010-10-15 Univ Utah Am n-terminus trunkierte isoformen von zyklischen phosphodiesterasen pde3a
US20050191627A1 (en) 2001-09-28 2005-09-01 Incyte Corporation Enzymes
ATE348104T1 (de) * 2001-10-29 2007-01-15 Univ Mcgill Azyklische linker enthaltende oligonukleotide und deren verwendungen
US7060498B1 (en) 2001-11-28 2006-06-13 Genta Salus Llc Polycationic water soluble copolymer and method for transferring polyanionic macromolecules across biological barriers
US7141540B2 (en) 2001-11-30 2006-11-28 Genta Salus Llc Cyclodextrin grafted biocompatible amphilphilic polymer and methods of preparation and use thereof
AU2002357860A1 (en) 2001-12-14 2003-06-30 Incyte Genomics, Inc. Enzymes
JP2005517433A (ja) 2002-02-20 2005-06-16 サーナ・セラピューティクス・インコーポレイテッド 短干渉核酸(siNA)を用いるTNFおよびTNFレセプタースーパーファミリー遺伝子発現のRNA干渉媒介性阻害
JP2005517427A (ja) 2002-02-20 2005-06-16 サーナ・セラピューティクス・インコーポレイテッド 短干渉核酸(siNA)を用いるC型肝炎ウイルス(HCV)遺伝子発現のRNA干渉媒介性阻害
EP1442143A4 (en) 2002-02-20 2005-02-16 Sirna Therapeutics Inc INHIBITION OF BCL2 GENE EXPRESSION MEDIATED BY RNA INTERFERENCE USING siNA (SHORT INTERFERING NUCLEIC ACID)
JP2005517450A (ja) 2002-02-20 2005-06-16 サーナ・セラピューティクス・インコーポレイテッド 短干渉核酸(siNA)を用いるRNA干渉媒介性標的発見および標的評価
AU2003219833A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics Inc. RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US7935812B2 (en) 2002-02-20 2011-05-03 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc INTERFERENCE MEDIATION INHIBITION OF GENE RNA FROM MAP KINASE
AU2003213057A1 (en) 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Incoporated Rna interference mediated inhibition of checkpoint kinase-1 (chk-1) gene expression using short interfering nucleic acid
WO2003106476A1 (en) 2002-02-20 2003-12-24 Sirna Therapeutics, Inc Nucleic acid mediated inhibition of enterococcus infection and cytolysin toxin activity
AU2003213054A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF POLYCOMB GROUP PROTEIN EZH2 GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
AU2003219781A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc RNA INTERFERENCE MEDIATED INHIBITION OF PROLIFERATING CELL NUCLEAR ANTIGEN (PCNA) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP1432725A4 (en) 2002-02-20 2005-03-30 Sirna Therapeutics Inc RNA INTERFERENCE-MEDIATED INHIBITION OF TELOMERASEGENEXPRESSION WITH SHORT INTERFERING NUCLEIC ACID (SINA)
US8232383B2 (en) 2002-02-20 2012-07-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003210895A1 (en) 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Inc. Rna interference mediated inhibition of cyclin d1 gene expression using short interfering nucleic acid (sina)
CA2480308C (en) 2002-03-27 2011-10-04 Aegera Therapeutics Inc. Antisense iap nucleobase oligomers and uses thereof
EP2333062A1 (en) * 2002-07-10 2011-06-15 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA-interference by single-stranded RNA molecules
DK1527176T4 (en) 2002-08-05 2017-07-03 Silence Therapeutics Gmbh ADDITIONAL NEW FORMS OF INTERFERRING RNA MOLECULES
US8729036B2 (en) * 2002-08-07 2014-05-20 University Of Massachusetts Compositions for RNA interference and methods of use thereof
US20040029275A1 (en) * 2002-08-10 2004-02-12 David Brown Methods and compositions for reducing target gene expression using cocktails of siRNAs or constructs expressing siRNAs
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8133903B2 (en) 2003-10-21 2012-03-13 Los Angeles Biomedical Research Institute at Harbor—UCLA Medical Center Methods of use of inhibitors of phosphodiesterases and modulators of nitric oxide, reactive oxygen species, and metalloproteinases in the treatment of peyronie's disease, arteriosclerosis and other fibrotic diseases
WO2004044140A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceticals, Inc. 2’-substituted oligomeric compounds and compositions for use in gene modulations
US7816337B2 (en) 2003-02-18 2010-10-19 Roche Madison Inc. Reversible attachment of a membrane active polymer to a polynucleotide
US6977223B2 (en) 2003-03-07 2005-12-20 Massachusetts Institute Of Technology Three dimensional microfabrication
EP1608733B1 (en) 2003-04-02 2011-12-07 Dharmacon, Inc. Modified polynucleotides for use in rna interference
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
JP2007505606A (ja) 2003-09-16 2007-03-15 サーナ・セラピューティクス・インコーポレイテッド 低分子干渉核酸(siNA)を使用したC型肝炎ウィルス(HCV)発現のRNA干渉媒介性抑制
BRPI0413930A (pt) * 2003-09-18 2006-10-24 Isis Pharmaceuticals Inc composto oligomérico ou sal farmaceuticamente aceitável deste, composição farmacêutica ou veterinária, métodos para inibir a expressão de eif4e em uma célula, tecido ou órgão, para diminuir a proliferação de uma célula em que eif4e é expressado, e para prevenir ou tratar uma condição ou doença métodos para prevenir ou diminuir a angiogênese, e o crescimento de tumor em um paciente, oligonucleotìdeo de anti-sentido, composição farmacêutica ou veterinária, e, uso de um composto oligomérico ou sal farmaceuticamente aceitável deste
US7943179B2 (en) 2003-09-23 2011-05-17 Massachusetts Institute Of Technology pH triggerable polymeric particles
WO2005030787A2 (en) 2003-09-29 2005-04-07 Topigen Pharmaceutique Inc. Oligonucleotide compositions and methods for treating disease including inflammatory conditions
EP1682661A2 (en) 2003-10-23 2006-07-26 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina)
EP1675948A2 (en) 2003-10-23 2006-07-05 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
WO2005060697A2 (en) 2003-12-19 2005-07-07 Chiron Corporation Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
US7858769B2 (en) 2004-02-10 2010-12-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional siNA)
WO2005089268A2 (en) * 2004-03-15 2005-09-29 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of rna by rnase h
MXPA06012076A (es) 2004-04-20 2007-01-25 Nastech Pharm Co Metodos y composiciones para mejorar el suministro de arn bicatenario o un acido nucleico hibrido bicatenario para regular la expresion genetica en celulas de mamifero.
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
ATE450251T1 (de) 2004-05-17 2009-12-15 Tekmira Pharmaceuticals Corp Liposomale formulierungen mit dihydrosphingomyelin und verfahren zu ihrer verwendung
US20060019258A1 (en) 2004-07-20 2006-01-26 Illumina, Inc. Methods and compositions for detection of small interfering RNA and micro-RNA
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
DK2230304T3 (da) * 2005-01-07 2012-07-16 Alnylam Pharmaceuticals Inc IRNA-modulering af RSV og terapeutiske anvendelser deraf
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2007086883A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
EP2157182A3 (en) 2005-03-08 2012-04-25 Qiagen GmbH Modified short interfering RNA
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20090176725A1 (en) 2005-08-17 2009-07-09 Sirna Therapeutics Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
CA2619876A1 (en) 2005-08-17 2007-02-22 Sirna Therapeutics, Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
US20080311040A1 (en) * 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
US8841436B2 (en) * 2007-03-15 2014-09-23 University Hospitals Cleveland Medical Center Screening, diagnosing, treating and prognosis of pathophysiologic status by RNA regulation
MX2009012568A (es) 2007-05-22 2009-12-08 Mdrna Inc Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico.
CN101821390A (zh) * 2007-06-14 2010-09-01 米尔克斯治疗有限责任公司 用于调节靶rna活性的寡核苷酸
US8614309B2 (en) * 2007-10-03 2013-12-24 Quark Pharmaceuticals, Inc. Double-stranded RNA directed to CASP2 and methods of use thereof
CA2708171C (en) 2007-12-04 2018-02-27 Alnylam Pharmaceuticals, Inc. Folate conjugates
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
US20110218334A1 (en) * 2008-07-11 2011-09-08 Alnylam Pharmaceuticals, Inc. PHOSPHOROTHIOATE OLIGONUCLEOTIDES AND NON-NUCLEOSIDIC PHOSPHOROTHIOATES AS DELIVERY AGENTS FOR iRNA AGENTS
JP2012502040A (ja) * 2008-09-02 2012-01-26 アルニラム ファーマスーティカルズ インコーポレイテッド 三リン酸塩オリゴヌクレオチドの合成方法および誘導体
EP2358398A2 (en) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
MX2011008383A (es) 2009-02-10 2011-09-06 Idera Pharmaceuticals Inc Agonistas basados en acido ribonucleico sintetico de receptor 7 tipo toll.
EP2669290A1 (en) * 2009-03-02 2013-12-04 Alnylam Pharmaceuticals Inc. Nucleic Acid Chemical Modifications
WO2011028550A1 (en) 2009-08-24 2011-03-10 Merck Sharp & Dohme Corp. Segmented micro rna mimetics
PL2470656T3 (pl) * 2009-08-27 2015-08-31 Idera Pharmaceuticals Inc Kompozycja do hamowania ekspresji genów i jej zastosowanie
WO2011133876A2 (en) * 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
EP2609198B8 (en) * 2010-08-24 2018-03-28 Sirna Therapeutics, Inc. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
WO2012068187A1 (en) 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
DK3366775T4 (da) 2011-11-18 2025-10-27 Alnylam Pharmaceuticals Inc Modificerede rnai-midler
EP3358013B1 (en) 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions

Also Published As

Publication number Publication date
US10584335B2 (en) 2020-03-10
JP2022037070A (ja) 2022-03-08
KR20130137160A (ko) 2013-12-16
JP2023100832A (ja) 2023-07-19
US9845466B2 (en) 2017-12-19
JP2017079776A (ja) 2017-05-18
EP3372684A1 (en) 2018-09-12
EP2609198B8 (en) 2018-03-28
JP2013535982A (ja) 2013-09-19
EP2609198A1 (en) 2013-07-03
JP2020074788A (ja) 2020-05-21
US20160222381A1 (en) 2016-08-04
CN103140582A (zh) 2013-06-05
US20180080024A1 (en) 2018-03-22
JP7527437B2 (ja) 2024-08-02
US9243246B2 (en) 2016-01-26
US20130171242A1 (en) 2013-07-04
EP2609198B1 (en) 2018-02-21
EP2609198A4 (en) 2014-11-19
EP3372684B1 (en) 2020-10-07
WO2012027206A1 (en) 2012-03-01

Similar Documents

Publication Publication Date Title
JP7527437B2 (ja) 内部非核酸スペーサーを含む一本鎖RNAi剤
JP5876637B2 (ja) ニックまたはギャップの入った核酸分子およびそれらの使用
JP2010503382A (ja) 低分子干渉核酸(siNA)を用いたプロタンパク質転換酵素サブチリシンケクシン9(PCSK9)遺伝子発現のRNA干渉媒介性阻害
KR20080066987A (ko) siRNA에 대한 전달 운반체로서의 펩티드-다이서 기질RNA 접합체들
JP2008514647A (ja) 二本鎖リボ核酸によって炎症性疾患を治療する方法
WO2008109369A2 (en) Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
JP2010519908A (ja) Hif1a遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519907A (ja) Vegfファミリー遺伝子の発現を抑制するための核酸化合物およびその使用
JP2011530289A (ja) Plk1遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519906A (ja) Erbbファミリー遺伝子の発現を抑制するための核酸化合物およびその使用
JP2012509688A (ja) 短鎖干渉核酸(siNA)を用いた上皮ナトリウムチャンネル(ENaC)遺伝子発現のRNA干渉媒介阻害
JP2010519911A (ja) Myc遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519912A (ja) Ras遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519905A (ja) Akt遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519910A (ja) Bcl2遺伝子の発現を抑制するための核酸化合物およびその使用
KR20080044909A (ko) 세포 내로 리보핵산의 전달을 위한 약학 조성물
WO2008109356A1 (en) Nucleic acid compounds for inhibiting tnfsf13b gene expression and uses thereof
WO2025255368A2 (en) Microrna 29b mimics
CN120813691A (zh) Reversir分子及其使用方法

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20140818

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20150901

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20151015

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160229

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20160524

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20160823

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20161222

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20170105

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: 20170207

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20170303

R150 Certificate of patent or registration of utility model

Ref document number: 6106085

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

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