CN104126010B - 嵌合的双链核酸 - Google Patents

嵌合的双链核酸 Download PDF

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CN104126010B
CN104126010B CN201280062110.XA CN201280062110A CN104126010B CN 104126010 B CN104126010 B CN 104126010B CN 201280062110 A CN201280062110 A CN 201280062110A CN 104126010 B CN104126010 B CN 104126010B
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nucleic acid
nucleotides
acid strand
strand
double
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CN104126010A (zh
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横田隆德
仁科一隆
小比贺聪
水泽英洋
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Tokyo Medical and Dental University NUC
University of Osaka NUC
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Tokyo Medical and Dental University NUC
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    • 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
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CN201280062110.XA 2011-12-16 2012-12-17 嵌合的双链核酸 Active CN104126010B (zh)

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JP2011275488 2011-12-16
JP2011-275488 2011-12-16
PCT/JP2012/083180 WO2013089283A1 (en) 2011-12-16 2012-12-17 Chimeric double-stranded nucleic acid

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EP (1) EP2791335B1 (cg-RX-API-DMAC7.html)
JP (7) JP6112569B2 (cg-RX-API-DMAC7.html)
CN (2) CN104126010B (cg-RX-API-DMAC7.html)
AU (1) AU2012353330B2 (cg-RX-API-DMAC7.html)
CA (2) CA3106312C (cg-RX-API-DMAC7.html)
ES (1) ES2707232T3 (cg-RX-API-DMAC7.html)
IN (1) IN2014CN04590A (cg-RX-API-DMAC7.html)
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Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG177564A1 (en) 2009-07-06 2012-02-28 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
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
CN104126010B (zh) * 2011-12-16 2021-04-06 国立大学法人东京医科齿科大学 嵌合的双链核酸
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Wave Life Sciences Japan 不斉補助基
JP6440170B2 (ja) * 2013-03-01 2018-12-19 国立大学法人 東京医科歯科大学 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
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
AU2014282666A1 (en) * 2013-06-16 2016-01-07 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
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts 十分に安定化された非対称sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017053999A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US20180223280A1 (en) * 2015-10-23 2018-08-09 Rena Therapeutics Inc. Nucleic acid complex
US20180228830A1 (en) * 2015-10-23 2018-08-16 Rena Therapeutics Inc. Nucleic acid complex having at least one bulge structure
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
AU2017211461B2 (en) * 2016-01-26 2023-01-12 Nissan Chemical Corporation Single-stranded oligonucleotide
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2018056442A1 (ja) * 2016-09-23 2018-03-29 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
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
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
AU2018307294B2 (en) 2017-07-26 2025-04-17 Nissan Chemical Corporation Single-stranded oligonucleotide
JP2021506239A (ja) 2017-12-14 2021-02-22 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. 複合アンチセンス化合物及びその使用
WO2019167995A1 (ja) 2018-02-28 2019-09-06 国立大学法人東京医科歯科大学 虚血病変部位特異的な遺伝子治療法
WO2019181946A1 (ja) 2018-03-19 2019-09-26 国立大学法人東京医科歯科大学 毒性が軽減した核酸
WO2019182294A1 (ko) * 2018-03-19 2019-09-26 주식회사 케이티 비디오 신호 처리 방법 및 장치
AU2019237599A1 (en) 2018-03-20 2020-11-12 Nissan Chemical Corporation Antisense oligonucleotide having reduced toxicity
EP3770257A4 (en) * 2018-03-22 2022-03-09 National University Corporation Tokyo Medical and Dental University BHS-PASSING LIPID LIGAND OF A HETERONUCLEIC 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
WO2020209285A1 (ja) 2019-04-08 2020-10-15 国立大学法人東京医科歯科大学 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
US20240117346A1 (en) * 2019-10-11 2024-04-11 National University Corporation Tokyo Medical And Dental University Modified hetero nucleic acids
EP4090744A4 (en) * 2020-01-17 2024-04-10 Anastasia Khvorova UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS
CA3169474A1 (en) 2020-01-31 2021-08-05 Sanwa Kagaku Kenkyusho Co., Ltd. Antisense oligonucleotide of atn1
JPWO2021177418A1 (cg-RX-API-DMAC7.html) 2020-03-04 2021-09-10
JPWO2021187392A1 (cg-RX-API-DMAC7.html) * 2020-03-16 2021-09-23
CN115968374A (zh) 2020-06-15 2023-04-14 Liid制药股份有限公司 交联型核苷及核苷酸
US20240002853A1 (en) 2020-11-23 2024-01-04 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 ????
CN117412773A (zh) 2021-05-31 2024-01-16 瑞纳治疗公司 配体结合核酸复合物
JPWO2023022229A1 (cg-RX-API-DMAC7.html) * 2021-08-19 2023-02-23
EP4389893A4 (en) 2021-08-21 2025-09-10 Takeda Pharmaceuticals Co PEPTIDE-DRUG CONJUGATE BINDING HUMAN TRANSFERRIN RECEPTOR
US20250179486A1 (en) 2021-09-15 2025-06-05 National University Corporation Tokyo Medical And Dental University Heteronucleic acid including 2'-modified nucleoside
WO2023064937A1 (en) * 2021-10-14 2023-04-20 Arizona Board Of Regents On Behalf Of Arizona State University Programmable delivery platforms for rna structures
JPWO2023080159A1 (cg-RX-API-DMAC7.html) 2021-11-02 2023-05-11
US20250297249A1 (en) 2021-12-07 2025-09-25 Liid Pharmaceuticals, Inc. Antisense oligonucleotide complex
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005113571A2 (en) * 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methods for the delivery of oligomeric compounds

Family Cites Families (31)

* 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
AU2002249967A1 (en) 2001-11-12 2003-05-26 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
AU2003273336A1 (en) * 2002-09-18 2004-04-08 Isis Pharmaceuticals, Inc. Efficient reduction of target rna's by single- and double-stranded oligomeric compounds
EP2336318B1 (en) * 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
US7427672B2 (en) 2003-08-28 2008-09-23 Takeshi Imanishi Artificial nucleic acids of n-o bond crosslinkage type
CN100558893C (zh) * 2003-09-18 2009-11-11 Isis药物公司 eIF4E表达的调节
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
WO2006074108A2 (en) * 2004-12-30 2006-07-13 Hauser Todd M Compositions and methods for modulating gene expression using self-protected oligonucleotides
EP2015758B1 (en) 2006-05-05 2014-04-02 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression apob
US8673548B2 (en) 2006-08-10 2014-03-18 Oncotherapy Science, Inc. Genes and polypeptides relating to breast cancers
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
EP2076597A2 (en) 2006-10-09 2009-07-08 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
NZ587178A (en) * 2008-02-08 2011-11-25 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
DK3252068T3 (da) * 2009-10-12 2025-08-25 Larry J Smith Metoder og sammensætninger til modulering af genekspression ved anvendelse af oligonukleotidbaserede lægemidler administreret in vivo eller in vitro
CN104126010B (zh) 2011-12-16 2021-04-06 国立大学法人东京医科齿科大学 嵌合的双链核酸
EP3004347B1 (en) * 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005113571A2 (en) * 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methods for the delivery of oligomeric compounds

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
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";Zhou et al;《J. Org. Chem》;20081204;摘要,表1,图3 *

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