CN112386605A - 嵌合的双链核酸 - Google Patents

嵌合的双链核酸 Download PDF

Info

Publication number
CN112386605A
CN112386605A CN202011292547.5A CN202011292547A CN112386605A CN 112386605 A CN112386605 A CN 112386605A CN 202011292547 A CN202011292547 A CN 202011292547A CN 112386605 A CN112386605 A CN 112386605A
Authority
CN
China
Prior art keywords
nucleic acid
nucleotides
modified
nucleotide analogs
strand
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.)
Pending
Application number
CN202011292547.5A
Other languages
English (en)
Chinese (zh)
Inventor
横田隆德
仁科一隆
小比贺聪
水泽英洋
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.)
Tokyo Medical and Dental University NUC
University of Osaka NUC
Original Assignee
Osaka University NUC
Tokyo Medical and Dental University NUC
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 Osaka University NUC, Tokyo Medical and Dental University NUC filed Critical Osaka University NUC
Publication of CN112386605A publication Critical patent/CN112386605A/zh
Pending 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
    • C12N15/113—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
    • 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
    • C12N2310/50—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202011292547.5A 2011-12-16 2012-12-17 嵌合的双链核酸 Pending CN112386605A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2011-275488 2011-12-16
JP2011275488 2011-12-16
CN201280062110.XA CN104126010B (zh) 2011-12-16 2012-12-17 嵌合的双链核酸

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201280062110.XA Division CN104126010B (zh) 2011-12-16 2012-12-17 嵌合的双链核酸

Publications (1)

Publication Number Publication Date
CN112386605A true CN112386605A (zh) 2021-02-23

Family

ID=47559626

Family Applications (2)

Application Number Title Priority Date Filing Date
CN202011292547.5A Pending CN112386605A (zh) 2011-12-16 2012-12-17 嵌合的双链核酸
CN201280062110.XA Active CN104126010B (zh) 2011-12-16 2012-12-17 嵌合的双链核酸

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN201280062110.XA Active CN104126010B (zh) 2011-12-16 2012-12-17 嵌合的双链核酸

Country Status (9)

Country Link
US (6) US9816089B2 (enExample)
EP (1) EP2791335B1 (enExample)
JP (7) JP6112569B2 (enExample)
CN (2) CN112386605A (enExample)
AU (1) AU2012353330B2 (enExample)
CA (2) CA3106312C (enExample)
ES (1) ES2707232T3 (enExample)
IN (1) IN2014CN04590A (enExample)
WO (1) WO2013089283A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108884462A (zh) * 2016-01-26 2018-11-23 日产化学株式会社 单链寡核苷酸

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
JP6128529B2 (ja) 2011-07-19 2017-05-17 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． 官能化核酸の合成のための方法
CN112386605A (zh) * 2011-12-16 2021-02-23 国立大学法人东京医科齿科大学 嵌合的双链核酸
US9598458B2 (en) 2012-07-13 2017-03-21 Wave Life Sciences Japan, Inc. Asymmetric auxiliary group
AU2014222150A1 (en) * 2013-03-01 2015-09-10 National University Corporation Tokyo Medical And Dental University Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
WO2014148620A1 (ja) * 2013-03-21 2014-09-25 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
US10190117B2 (en) * 2013-06-16 2019-01-29 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
WO2015105083A1 (ja) * 2014-01-07 2015-07-16 塩野義製薬株式会社 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
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US11116843B2 (en) 2015-09-25 2021-09-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2017068791A1 (ja) * 2015-10-23 2017-04-27 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
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
JP7043078B2 (ja) * 2016-09-23 2022-03-29 国立大学法人 東京医科歯科大学 血液脳関門通過型ヘテロ２本鎖核酸
WO2018062510A1 (ja) 2016-09-29 2018-04-05 国立大学法人東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体
CA3052801A1 (en) * 2017-02-06 2018-08-09 Nissan Chemical Corporation Single-stranded oligonucleotide
US11638717B2 (en) 2017-03-29 2023-05-02 Shionogi & Co., Ltd. Complex of nucleic acid medicine and multibranched lipid
EP3604531A4 (en) * 2017-03-31 2020-12-23 Aichi Medical University NUCLEIC ACID ANTISENS FOR INHIBITION OF CHONDROITIN SULPHATE BIOSYNTHESIS
JP6974872B2 (ja) * 2017-06-30 2021-12-01 国立大学法人 東京医科歯科大学 ヘテロ二本鎖型ａｎｔｉｍｉＲ
CA3070809A1 (en) 2017-07-26 2019-01-31 Nissan Chemical Corporation Single-stranded oligonucleotide
US11725208B2 (en) 2017-12-14 2023-08-15 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US11674141B2 (en) 2018-02-28 2023-06-13 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
CA3094303A1 (en) 2018-03-20 2019-09-26 Tokyo Institute Of Technology Antisense oligonucleotide reduced in toxicity
EP3770257B1 (en) * 2018-03-22 2026-01-28 Institute Of Science Tokyo Bbb-passing lipid ligand of hetero nucleic acid
CN113614232A (zh) * 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
JP7687682B2 (ja) * 2019-04-08 2025-06-03 国立大学法人東京科学大学 筋疾患治療用医薬組成物
EP4005602A4 (en) 2019-07-30 2024-06-12 Shionogi & Co., Ltd NUCLEIC ACID AGENT TARGETED AGAINST MURF1
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
CN114929876A (zh) 2020-01-31 2022-08-19 株式会社三和化学研究所 Atn1的反义寡核苷酸
JPWO2021177418A1 (enExample) 2020-03-04 2021-09-10
WO2021187392A1 (ja) 2020-03-16 2021-09-23 国立大学法人東京医科歯科大学 モルホリノ核酸を含むヘテロ核酸
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
JP7751302B2 (ja) 2020-06-15 2025-10-08 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
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
US20240141414A1 (en) * 2021-02-26 2024-05-02 Northwestern University Cell-free biosensors with dna strand displacement circuits
WO2022250050A1 (ja) 2021-05-25 2022-12-01 国立大学法人東京医科歯科大学 ｓｃｐＢＮＡ又はＡｍＮＡを含むヘテロ核酸
US20240279655A1 (en) 2021-05-31 2024-08-22 Rena Therapeutics Inc. Ligand-binding nucleic acid complex
EP4389891A4 (en) * 2021-08-19 2025-10-29 Univ Nat Corp Tokyo Medical & Dental MODIFIED HETERONUCLEIC ACID CONTAINING MORPHOLINO NUCLEIC ACID
JPWO2023026994A1 (enExample) 2021-08-21 2023-03-02
WO2023042876A1 (ja) 2021-09-15 2023-03-23 国立大学法人東京医科歯科大学 ２'－修飾ヌクレオシドを含むヘテロ核酸
EP4415827A4 (en) * 2021-10-14 2025-12-03 Univ Arizona State PROGRAMMABLE DISTRIBUTION PLATFORMS FOR RNA STRUCTURES
JPWO2023080159A1 (enExample) 2021-11-02 2023-05-11
JP7412668B2 (ja) * 2021-12-07 2024-01-15 リードファーマ株式会社 アンチセンスオリゴヌクレオチド複合体
WO2024071099A1 (ja) 2022-09-29 2024-04-04 国立大学法人東京医科歯科大学 ５'－シクロプロピレン修飾を含む核酸分子
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040137471A1 (en) * 2002-09-18 2004-07-15 Timothy Vickers Efficient reduction of target RNA's by single-and double-stranded oligomeric compounds
US20080199960A1 (en) * 2004-05-13 2008-08-21 Juliano Rudolph L Methods for the Delivery of Oligomeric Compounds

Family Cites Families (30)

* 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
EP2253639A1 (en) 1997-09-12 2010-11-24 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
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
AU2002249967A1 (en) * 2001-11-12 2003-05-26 Epiclone, Inc. Gene silencing using sense dna and antisense rna hybrid constructs
AU2003247521A1 (en) * 2002-06-26 2004-01-19 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
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
ES2382807T3 (es) 2003-08-28 2012-06-13 Takeshi Imanishi Nuevos ácidos nucleicos artificiales del tipo de enlace N-O con reticulación
PE20050469A1 (es) * 2003-09-18 2005-09-10 Lilly Co Eli Modulacion de la expresion de eif4e
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
EP1838875A4 (en) * 2004-12-30 2010-08-25 Todd M Hauser COMPOSITIONS AND METHODS FOR MODULATING GENE EXPRESSION USING SELF-PROTECTED OLIGONUCLEOTIDES
ATE514777T1 (de) 2006-05-05 2011-07-15 Isis Pharmaceuticals Inc Verbindungen und verfahren zur modulation von genexpression
KR20090064378A (ko) * 2006-08-10 2009-06-18 온코세라피 사이언스 가부시키가이샤 유방암 관련 유전자 및 폴리펩티드
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
ES2603379T3 (es) 2006-10-09 2017-02-27 Roche Innovation Center Copenhagen A/S Compuestos antagonistas de ARN para la modulación de 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
US8461124B2 (en) * 2007-03-15 2013-06-11 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2′,4′-carbocyclic ribo-thymidines for the treatment of infections and cancer
CN101980726A (zh) * 2008-02-08 2011-02-23 普罗森那控股公司 治疗与dna重复不稳定性相关的遗传病症的方法和装置
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
EP3252068B1 (en) * 2009-10-12 2025-07-02 Larry J. Smith Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
CN112386605A (zh) * 2011-12-16 2021-02-23 国立大学法人东京医科齿科大学 嵌合的双链核酸
CA2913499A1 (en) * 2013-05-30 2014-12-04 National University Corporation Tokyo Medical And Dental University Double-stranded agents for delivering therapeutic oligonucleotides

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040137471A1 (en) * 2002-09-18 2004-07-15 Timothy Vickers Efficient reduction of target RNA's by single-and double-stranded oligomeric compounds
US20080199960A1 (en) * 2004-05-13 2008-08-21 Juliano Rudolph L Methods for the Delivery of Oligomeric Compounds

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CHUANZHENG ZHOU等: "Fine Tuning of Electrostatics around the Internucleotidic Phosphate through Incorporation of Modified 2′,4′-Carbocyclic-LNAs and -ENAs Leads to Significant Modulation of Antisense Properties", 《J. ORG. CHEM.》, vol. 74, no. 1, pages 118 - 134, XP055055373, DOI: 10.1021/jo8016742 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108884462A (zh) * 2016-01-26 2018-11-23 日产化学株式会社 单链寡核苷酸
CN108884462B (zh) * 2016-01-26 2022-11-25 日产化学株式会社 单链寡核苷酸

Also Published As

Publication number Publication date
CA3106312A1 (en) 2013-06-20
US10337006B2 (en) 2019-07-02
CA2859581C (en) 2022-03-22
JP2022171811A (ja) 2022-11-11
WO2013089283A1 (en) 2013-06-20
JP6112569B2 (ja) 2017-04-12
US20180320181A1 (en) 2018-11-08
JP2022000426A (ja) 2022-01-04
US12344841B2 (en) 2025-07-01
JP6638923B2 (ja) 2020-02-05
JP2017140031A (ja) 2017-08-17
US11034955B2 (en) 2021-06-15
US20180073024A1 (en) 2018-03-15
US20190270996A1 (en) 2019-09-05
JP7174384B2 (ja) 2022-11-17
ES2707232T3 (es) 2019-04-03
CN104126010A (zh) 2014-10-29
EP2791335A1 (en) 2014-10-22
CN104126010B (zh) 2021-04-06
US10329567B2 (en) 2019-06-25
IN2014CN04590A (enExample) 2015-09-18
US20210340540A1 (en) 2021-11-04
JP2020039364A (ja) 2020-03-19
JP6416301B2 (ja) 2018-10-31
US9816089B2 (en) 2017-11-14
US20140302603A1 (en) 2014-10-09
JP7423015B2 (ja) 2024-01-29
JP6960123B2 (ja) 2021-11-05
JP2018203779A (ja) 2018-12-27
US20240158790A1 (en) 2024-05-16
AU2012353330A1 (en) 2014-07-31
CA3106312C (en) 2025-09-23
EP2791335B1 (en) 2018-11-14
JP2015502134A (ja) 2015-01-22
JP2021011501A (ja) 2021-02-04
AU2012353330B2 (en) 2018-04-19
CA2859581A1 (en) 2013-06-20

Similar Documents

Publication Publication Date Title
CN104126010B (zh) 嵌合的双链核酸
JP6440170B2 (ja) キメラ一本鎖アンチセンスポリヌクレオチドおよび二本鎖アンチセンス剤
WO2014203518A1 (en) Double-stranded antisense nucleic acid with exon-skipping effect
AU2010258875A1 (en) Chemical modification motifs for miRNA inhibitors and mimetics
HK40084771B (zh) 用於抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
WO2020262555A1 (ja) 核酸医薬副作用軽減剤、該核酸医薬副作用軽減剤を含む医薬組成物、並びに核酸医薬の副作用惹起性を軽減する方法

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination