CN104011209A - 病毒基因表达的抑制 - Google Patents

病毒基因表达的抑制 Download PDF

Info

Publication number
CN104011209A
CN104011209A CN201280053081.0A CN201280053081A CN104011209A CN 104011209 A CN104011209 A CN 104011209A CN 201280053081 A CN201280053081 A CN 201280053081A CN 104011209 A CN104011209 A CN 104011209A
Authority
CN
China
Prior art keywords
sirna
modified
nucleotide
guanidinopropyl
nucleotide molecule
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
CN201280053081.0A
Other languages
English (en)
Chinese (zh)
Inventor
帕特里克·阿巴思诺特
贾斯廷·希恩
阿布杜拉·伊利
穆萨·马里亚尼
约兰塔·布瑞恩斯卡
詹尼弗·多诺费奥
马克西米利安C.R.·布夫
约阿希姆W.·恩格斯
斯蒂芬·贝恩哈特
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.)
Goethe Universitaet Frankfurt am Main
University of the Witwatersrand, Johannesburg
Original Assignee
Goethe Universitaet Frankfurt am Main
University of the Witwatersrand, Johannesburg
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 Goethe Universitaet Frankfurt am Main, University of the Witwatersrand, Johannesburg filed Critical Goethe Universitaet Frankfurt am Main
Publication of CN104011209A publication Critical patent/CN104011209A/zh
Pending legal-status Critical Current

Links

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
    • C12N15/1131—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 against viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527—Other alkyl chain
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201280053081.0A 2011-10-28 2012-10-26 病毒基因表达的抑制 Pending CN104011209A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ZA201107890 2011-10-28
ZA2011/07890 2011-10-28
PCT/IB2012/055915 WO2013061295A1 (en) 2011-10-28 2012-10-26 Inhibition of viral gene expression

Publications (1)

Publication Number Publication Date
CN104011209A true CN104011209A (zh) 2014-08-27

Family

ID=47326248

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201280053081.0A Pending CN104011209A (zh) 2011-10-28 2012-10-26 病毒基因表达的抑制

Country Status (9)

Country Link
US (1) US20140350080A1 (enExample)
EP (1) EP2771466A1 (enExample)
CN (1) CN104011209A (enExample)
AP (1) AP2014007650A0 (enExample)
BR (1) BR112014010134A2 (enExample)
CA (1) CA2853609A1 (enExample)
IN (1) IN2014CN03921A (enExample)
RU (1) RU2014121304A (enExample)
WO (1) WO2013061295A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110678447A (zh) * 2017-06-16 2020-01-10 卫材R&D管理有限公司 经修饰的核酸单体化合物及寡核酸类似物

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110257379B (zh) 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
CN107849567B (zh) * 2015-06-26 2024-07-23 苏州瑞博生物技术股份有限公司 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用
US20170137821A1 (en) 2015-07-17 2017-05-18 Arcturus Therapeutics, Inc. Molecules and agents for treating hepatitis b virus
CN108136206B (zh) 2015-07-17 2021-10-15 阿克丘勒斯治疗公司 抗乙型肝炎病毒的组合物和药剂及其用途
JP7360716B2 (ja) * 2017-12-01 2023-10-13 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物および複合体ならびに調製方法と使用
US20210395751A1 (en) * 2018-10-31 2021-12-23 The University Of Sydney Compositions and methods for treating viral infections
JP7507495B2 (ja) 2018-12-28 2024-06-28 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
CN118256498A (zh) 2019-05-22 2024-06-28 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1814614A (zh) * 2005-02-06 2006-08-09 中国人民解放军军事医学科学院毒物药物研究所 核酸、肽核酸衍生物及它们的用途
WO2009038266A1 (en) * 2007-09-17 2009-03-26 Mogam Biotechnology Research Institute Method for enhancing serum stability and lowering immune response of sirna down-regulating gene expression of hbv or hcv

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1814614A (zh) * 2005-02-06 2006-08-09 中国人民解放军军事医学科学院毒物药物研究所 核酸、肽核酸衍生物及它们的用途
WO2009038266A1 (en) * 2007-09-17 2009-03-26 Mogam Biotechnology Research Institute Method for enhancing serum stability and lowering immune response of sirna down-regulating gene expression of hbv or hcv

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
DALIBOR ODADZIC ET AL.: "Synthesis of 2′-O-modified adenosine building blocks and application for RNA interference", 《BIOORGANIC & MEDICINAL CHEMISTRY》, vol. 16, no. 1, 15 September 2007 (2007-09-15), pages 518 - 529 *
JOLANTA BRZEZINSKA ET AL.: "Synthesis of 2′-O-guanidinopropyl-modified nucleoside phosphoramidites and their incorporation into siRNAs targeting hepatitis B virus", 《BIOORGANIC & MEDICINAL CHEMISTRY》, vol. 20, 21 December 2011 (2011-12-21), pages 1594 - 1606, XP055053033, DOI: 10.1016/j.bmc.2011.12.024 *
JUSTIN HEAN ET AL.: "Inhibition of hepatitis B virus replication in vivo using lipoplexes containing altritol-modified antiviral siRNAs", 《ARTIFICIAL DNA: PNA & XNA》, vol. 1, no. 1, 31 July 2010 (2010-07-31), pages 17 - 26, XP055073339 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110678447A (zh) * 2017-06-16 2020-01-10 卫材R&D管理有限公司 经修饰的核酸单体化合物及寡核酸类似物
CN110678447B (zh) * 2017-06-16 2023-12-12 卫材R&D管理有限公司 经修饰的核酸单体化合物及寡核酸类似物

Also Published As

Publication number Publication date
EP2771466A1 (en) 2014-09-03
WO2013061295A1 (en) 2013-05-02
AP2014007650A0 (en) 2014-05-31
US20140350080A1 (en) 2014-11-27
RU2014121304A (ru) 2015-12-10
CA2853609A1 (en) 2013-05-02
BR112014010134A2 (pt) 2019-09-24
IN2014CN03921A (enExample) 2015-09-04

Similar Documents

Publication Publication Date Title
AU2020244546B2 (en) Chiral control
JP7684373B2 (ja) オフターゲット効果が低下した修飾ｒｎａ剤
CN104011209A (zh) 病毒基因表达的抑制
EP1789553B1 (en) Oligonucleotides comprising a non-phosphate backbone linkage
AU2005325262B2 (en) Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
AU2005323437B2 (en) Oligonucleotides comprising a C5-modified pyrimidine
AU2022206704A1 (en) Nanoparticle formulations for delivery of nucleic acid complexes
JP5816556B2 (ja) 治療剤のためのｕｎａオリゴマー構造
US20110294869A1 (en) Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
Brzezinska et al. Synthesis of 2′-O-guanidinopropyl-modified nucleoside phosphoramidites and their incorporation into siRNAs targeting hepatitis B virus
CN119768186A (zh) 用于向cns组织递送治疗剂的脂质缀合物
US10022454B2 (en) Functionalized phosphorodiamites for therapeutic oligonucleotide synthesis
WO2025119303A1 (en) Abasic modification compound, gene modulating agents and the use thereof
WO2025223315A1 (zh) 一种用于肝脏特异性递送的多靶标双链rna偶联物及药物组合物
TW202538051A (zh) 寡核苷酸的遞送載體及其使用方法
Malek-Adamian Synthesis of Novel 2′, 4′-Modified Nucleoside Analogues and Their Properties in Oligonucleotides and Nanoparticle Development for Targeted Delivery to Glioblastoma Multiformes Drr-Expressing Cells
WO2025148770A1 (zh) 修饰的核苷酸单体及其用途
CN119119155A (zh) 修饰的核苷酸单体及其用途
Bogojeski Synthesis and characterization of novel self-delivering amino acid-RNA conjugates for the development of potent siRNA prodrugs

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C02 Deemed withdrawal of patent application after publication (patent law 2001)
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20140827