JP6112569B2 - キメラ２重鎖核酸 - Google Patents

キメラ２重鎖核酸 Download PDF

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Publication number
JP6112569B2
JP6112569B2 JP2014528740A JP2014528740A JP6112569B2 JP 6112569 B2 JP6112569 B2 JP 6112569B2 JP 2014528740 A JP2014528740 A JP 2014528740A JP 2014528740 A JP2014528740 A JP 2014528740A JP 6112569 B2 JP6112569 B2 JP 6112569B2
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nucleic acid
pharmaceutical composition
acid strand
nucleotide
nucleotides
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Japanese (ja)
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JP2015502134A (ja
JP2015502134A5 (2
Inventor
隆徳 横田
隆徳 横田
一隆 仁科
一隆 仁科
聡 小比賀
聡 小比賀
水澤　英洋
英洋 水澤
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Tokyo Medical and Dental University NUC
University of Osaka NUC
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Osaka University NUC
Tokyo Medical and Dental University NUC
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Publication of JP2015502134A5 publication Critical patent/JP2015502134A5/ja
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    • 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
    • C12N15/113—Non-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
    • 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
    • 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
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • 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
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/3513—Protein; Peptide
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2014528740A 2011-12-16 2012-12-17 キメラ２重鎖核酸 Active JP6112569B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2014528740A JP6112569B2 (ja) 2011-12-16 2012-12-17 キメラ２重鎖核酸

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
JP2011275488 2011-12-16
JP2011275488 2011-12-16
JP2014528740A JP6112569B2 (ja) 2011-12-16 2012-12-17 キメラ２重鎖核酸
PCT/JP2012/083180 WO2013089283A1 (en) 2011-12-16 2012-12-17 Chimeric double-stranded nucleic acid

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017044034A Division JP6416301B2 (ja) 2011-12-16 2017-03-08 キメラ２重鎖核酸

Publications (3)

Publication Number Publication Date
JP2015502134A JP2015502134A (ja) 2015-01-22
JP2015502134A5 JP2015502134A5 (2) 2016-03-31
JP6112569B2 true JP6112569B2 (ja) 2017-04-12

Family

ID=47559626

Family Applications (7)

Application Number Title Priority Date Filing Date
JP2014528740A Active JP6112569B2 (ja) 2011-12-16 2012-12-17 キメラ２重鎖核酸
JP2017044034A Active JP6416301B2 (ja) 2011-12-16 2017-03-08 キメラ２重鎖核酸
JP2018188022A Active JP6638923B2 (ja) 2011-12-16 2018-10-03 キメラ２重鎖核酸
JP2019224279A Active JP6960123B2 (ja) 2011-12-16 2019-12-12 キメラ２重鎖核酸
JP2020179738A Active JP7174384B2 (ja) 2011-12-16 2020-10-27 キメラ２重鎖核酸
JP2021136038A Pending JP2022000426A (ja) 2011-12-16 2021-08-24 キメラ２重鎖核酸
JP2022146970A Active JP7423015B2 (ja) 2011-12-16 2022-09-15 キメラ２重鎖核酸

Family Applications After (6)

Application Number Title Priority Date Filing Date
JP2017044034A Active JP6416301B2 (ja) 2011-12-16 2017-03-08 キメラ２重鎖核酸
JP2018188022A Active JP6638923B2 (ja) 2011-12-16 2018-10-03 キメラ２重鎖核酸
JP2019224279A Active JP6960123B2 (ja) 2011-12-16 2019-12-12 キメラ２重鎖核酸
JP2020179738A Active JP7174384B2 (ja) 2011-12-16 2020-10-27 キメラ２重鎖核酸
JP2021136038A Pending JP2022000426A (ja) 2011-12-16 2021-08-24 キメラ２重鎖核酸
JP2022146970A Active JP7423015B2 (ja) 2011-12-16 2022-09-15 キメラ２重鎖核酸

Country Status (9)

Country Link
US (6) US9816089B2 (2)
EP (1) EP2791335B1 (2)
JP (7) JP6112569B2 (2)
CN (2) CN104126010B (2)
AU (1) AU2012353330B2 (2)
CA (2) CA2859581C (2)
ES (1) ES2707232T3 (2)
IN (1) IN2014CN04590A (2)
WO (1) WO2013089283A1 (2)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016526529A (ja) * 2013-06-16 2016-09-05 国立大学法人 東京医科歯科大学 エクソンスキッピング効果を有する二本鎖アンチセンス核酸

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
JP6112569B2 (ja) * 2011-12-16 2017-04-12 国立大学法人 東京医科歯科大学 キメラ２重鎖核酸
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
WO2014132671A1 (en) * 2013-03-01 2014-09-04 National University Corporation Tokyo Medical And Dental University Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
JP6300212B2 (ja) * 2013-03-21 2018-03-28 国立大学法人 東京医科歯科大学 二重鎖核酸結合剤、当該結合剤−二重鎖核酸複合体、当該複合体を含有する医薬品組成物、及び、当該複合体の製造方法
JP6482475B2 (ja) * 2014-01-07 2019-03-13 レナセラピューティクス株式会社 アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド
US9714288B2 (en) 2014-09-30 2017-07-25 The Regents Of The University Of California Antisense compounds and uses thereof
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP2018528783A (ja) 2015-09-25 2018-10-04 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． コンジュゲートアンチセンス化合物及びその使用
JPWO2017068791A1 (ja) * 2015-10-23 2018-08-09 レナセラピューティクス株式会社 少なくとも１つのバルジ構造を有する核酸複合体
JPWO2017068790A1 (ja) * 2015-10-23 2018-08-09 レナセラピューティクス株式会社 核酸複合体
WO2017094745A1 (ja) * 2015-12-01 2017-06-08 国立大学法人大阪大学 核酸を含む経腸投与用組成物
WO2017131124A1 (ja) * 2016-01-26 2017-08-03 日産化学工業株式会社 一本鎖オリゴヌクレオチド
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
EP3517610A4 (en) 2016-09-23 2020-05-27 National University Corporation Tokyo Medical and Dental University THROUGH HETERODUPLEX NUCLEIC ACID THROUGH BLOOD BRAIN CABINET
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
AU2018215440B2 (en) 2017-02-06 2024-10-24 Nissan Chemical Corporation Single-stranded oligonucleotide
EP3603648B1 (en) 2017-03-29 2025-09-17 Shionogi & Co., Ltd Complex of nucleic acid medicine and multibranched lipid
JP7141621B2 (ja) * 2017-03-31 2022-09-26 学校法人 愛知医科大学 コンドロイチン硫酸生合成を阻害するアンチセンス核酸
EP3647423A4 (en) * 2017-06-30 2021-03-24 National University Corporation Tokyo Medical and Dental University DOUBLE STRANDED HETERO-ANTI-MIRNA
BR112020001430A2 (pt) * 2017-07-26 2020-07-28 Nissan Chemical Corporation oligonucleotídeo de fita simples
US11725208B2 (en) 2017-12-14 2023-08-15 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3760209B1 (en) 2018-02-28 2023-11-22 National University Corporation Tokyo Medical and Dental University Ischemic-lesion-site-specific gene therapy
KR20190110043A (ko) * 2018-03-19 2019-09-27 주식회사 케이티 비디오 신호 처리 방법 및 장치
EP3769769A4 (en) * 2018-03-19 2022-05-04 National University Corporation Tokyo Medical and Dental University NUCLEIC ACID WITH REDUCED TOXICITY
BR112020018902A2 (pt) 2018-03-20 2021-01-26 Tokyo Institute Of Technology oligonucleotídeo antissenso com toxicidade reduzida
JP7262816B2 (ja) * 2018-03-22 2023-04-24 国立大学法人 東京医科歯科大学 ヘテロ核酸のｂｂｂ通過脂質リガンド
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
CN113677374A (zh) * 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
JP7692829B2 (ja) 2019-07-30 2025-06-16 塩野義製薬株式会社 Ｍｕｒｆ１を標的とする核酸医薬
EP4043565A4 (en) * 2019-10-11 2024-01-24 National University Corporation Tokyo Medical and Dental University MODIFIED HETERONUCLEIC ACID
EP4090744A4 (en) * 2020-01-17 2024-04-10 Anastasia Khvorova UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS
KR20220133925A (ko) 2020-01-31 2022-10-05 가부시키가이샤산와카가쿠켄큐쇼 Atn1의 안티센스 올리고뉴클레오티드
EP4116419A1 (en) 2020-03-04 2023-01-11 Nissan Chemical Corporation Antisense oligonucleotide of calm2
EP4123023A4 (en) * 2020-03-16 2024-07-17 National University Corporation Tokyo Medical and Dental University Heteronucleic acid containing morpholino nucleic acid
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
WO2021256297A1 (ja) 2020-06-15 2021-12-23 リードファーマ株式会社 架橋型ヌクレオシドおよびヌクレオチド
EP4247949A1 (en) 2020-11-23 2023-09-27 Alpha Anomeric SAS Nucleic acid duplexes
WO2022183102A1 (en) * 2021-02-26 2022-09-01 Northwestern University Cell-free biosensors with dna strand displacement circuits
EP4349986A4 (en) * 2021-05-25 2025-11-19 Univ Nat Corp Tokyo Medical & Dental HETERONUCLEIC ACID WITH A CONTENT OF ?????? OR ????
AU2022285344A1 (en) 2021-05-31 2023-12-14 Rena Therapeutics Inc. Ligand-bound nucleic acid complex
JPWO2023022229A1 (2) * 2021-08-19 2023-02-23
US20240390508A1 (en) 2021-08-21 2024-11-28 Takeda Pharmaceutical Company Limited Human transferrin receptor binding peptide-drug conjugate
EP4403191A4 (en) * 2021-09-15 2026-03-18 Univ Nat Corp Tokyo Medical & Dental HETERONUCLEIC ACID COMPRISING A 2'-MODIFIED NUCLEOSIDE
US20250000892A1 (en) * 2021-10-14 2025-01-02 Arizona Board Of Regents On Behalf Of Arizona State University Programmable delivery platforms for rna structures
EP4428238A1 (en) 2021-11-02 2024-09-11 Rena Therapeutics Inc. Ligand-bound nucleic acid complex
US20250297249A1 (en) 2021-12-07 2025-09-25 Liid Pharmaceuticals, Inc. Antisense oligonucleotide complex
EP4596694A1 (en) 2022-09-29 2025-08-06 Institute Of Science Tokyo Nucleic acid molecule containing 5'-cyclopropylene modification
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024228398A1 (ja) * 2023-05-02 2024-11-07 国立大学法人東京医科歯科大学 グアニン四重鎖構造を誘導するための核酸剤
WO2025047953A1 (ja) 2023-08-30 2025-03-06 株式会社Stratoimmune ＲａｓＧＲＰ４のアンチセンスオリゴヌクレオチド

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH09110894A (ja) * 1995-10-17 1997-04-28 Soyaku Gijutsu Kenkyusho:Kk ハイブリッドｄｎａ／ｒｎａオリゴヌクレオチド及び抗ウイルス剤
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
JP3781879B2 (ja) 1996-11-18 2006-05-31 武 今西 新規ヌクレオチド類縁体
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US20060074034A1 (en) * 2001-09-17 2006-04-06 Collins Douglas A Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof
FR2832154B1 (fr) * 2001-11-09 2007-03-16 Centre Nat Rech Scient Oligonucleotides inhibiteurs et leur utilisation pour reprimer specifiquement un gene
US20030104401A1 (en) 2001-11-12 2003-06-05 Epiclone, Inc. Gene silencing using sense DNA and antisense RNA hybrid constructs
WO2004003134A2 (en) * 2002-06-26 2004-01-08 Isis Pharmaceuticals, Inc. Antisense inhibition via rnase h-independent reduction in mrna
DE10240417A1 (de) 2002-09-02 2004-03-11 Avontec Gmbh Decoy-Oligonukleotid-Hemmung der CD40-Expression
EP1546344A4 (en) * 2002-09-18 2007-10-03 Isis Pharmaceuticals Inc EFFICIENT PRODUCTION OF TARGET RNAS USING SINGLE AND DOUBLE-STRONG OLIGOMER COMPOUNDS
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
EP2256201A3 (en) * 2003-09-18 2012-07-04 Isis Pharmaceuticals, Inc. Modulation of eIF4E expression
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
AU2011204898A1 (en) * 2004-04-09 2011-08-11 Biodelivery Sciences International, Inc Nucleotide-cochleate compositions and methods of use
US20080199960A1 (en) * 2004-05-13 2008-08-21 Juliano Rudolph L Methods for the Delivery of Oligomeric Compounds
US20090005332A1 (en) 2004-12-30 2009-01-01 Hauser Todd M Compositions and Methods for Modulating Gene Expression Using Self-Protected Oligonucleotides
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｐｃｓｋ９の発現を調節するための化合物および方法
JP5339291B2 (ja) * 2006-08-10 2013-11-13 オンコセラピー・サイエンス株式会社 乳癌に関連する遺伝子およびポリペプチド
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
CA2666191C (en) 2006-10-09 2017-07-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
US8093222B2 (en) * 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US7858592B2 (en) * 2007-02-26 2010-12-28 The Board Of Regents Of The University Of Texas System Interfering RNAs against the promoter region of P53
WO2008111908A1 (en) * 2007-03-15 2008-09-18 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2',4'- carbocyclic ribo-thymidines for the treatment of infections and cancer
WO2009099326A1 (en) * 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
EP2133425A1 (en) 2008-06-13 2009-12-16 Karin Mölling Method and compound for antiviral (HIV) therapy
US8541569B2 (en) * 2008-09-06 2013-09-24 Chemgenes Corporation Phosphoramidites for synthetic RNA in the reverse direction, efficient RNA synthesis and convenient introduction of 3'-end ligands, chromophores and modifications of synthetic RNA
AU2010306940A1 (en) * 2009-10-12 2012-06-07 Smith, Larry Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
JP6112569B2 (ja) 2011-12-16 2017-04-12 国立大学法人 東京医科歯科大学 キメラ２重鎖核酸
EP3004347B1 (en) * 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016526529A (ja) * 2013-06-16 2016-09-05 国立大学法人 東京医科歯科大学 エクソンスキッピング効果を有する二本鎖アンチセンス核酸

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