CN1744886A - 使用pei/单链寡核糖核苷酸复合物下调靶基因 - Google Patents

使用pei/单链寡核糖核苷酸复合物下调靶基因 Download PDF

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Publication number
CN1744886A
CN1744886A CNA2004800033048A CN200480003304A CN1744886A CN 1744886 A CN1744886 A CN 1744886A CN A2004800033048 A CNA2004800033048 A CN A2004800033048A CN 200480003304 A CN200480003304 A CN 200480003304A CN 1744886 A CN1744886 A CN 1744886A
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China
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gene
pei
target gene
ssrna
rna
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Pending
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CNA2004800033048A
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English (en)
Chinese (zh)
Inventor
J－C·博洛尼亚
J·哈尔
F·J-C·纳特
J·魏勒
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Novartis AG
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Novartis AG
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Publication of CN1744886A publication Critical patent/CN1744886A/zh
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    • 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
    • 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
    • A61K31/095—Sulfur, selenium, or tellurium compounds, e.g. thiols
    • A61K31/105—Persulfides
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
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    • 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
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Macromolecular Compounds Obtained By Forming Nitrogen-Containing Linkages In General (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
CNA2004800033048A 2003-01-31 2004-01-30 使用pei/单链寡核糖核苷酸复合物下调靶基因 Pending CN1744886A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44397703P 2003-01-31 2003-01-31
US60/443,977 2003-01-31

Publications (1)

Publication Number Publication Date
CN1744886A true CN1744886A (zh) 2006-03-08

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ID=32825395

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CNA2004800033048A Pending CN1744886A (zh) 2003-01-31 2004-01-30 使用pei/单链寡核糖核苷酸复合物下调靶基因

Country Status (7)

Country Link
US (1) US20060135453A1 (https=)
EP (1) EP1592412A1 (https=)
JP (1) JP2006517793A (https=)
CN (1) CN1744886A (https=)
BR (1) BRPI0407107A (https=)
CA (1) CA2514536A1 (https=)
WO (1) WO2004066989A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104349794A (zh) * 2012-06-08 2015-02-11 埃泽瑞斯公司 信使rna的肺递送
CN109562059A (zh) * 2016-07-15 2019-04-02 生物技术Rna制药有限公司 用于施用rna的制剂

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060030534A1 (en) * 2002-09-04 2006-02-09 Gabriele Dorn Treatment of neurological disorders by dsrna administration
EP2287301A3 (en) * 2004-08-31 2011-11-02 Sylentis S.A.U. Methods and compositions to inhibit P2X7 receptor expression
JP5802389B2 (ja) * 2007-12-13 2015-10-28 ポリプラス−トランスフェクション 合成ポリマーを用いた遺伝子サイレンシングに活性な核酸を送達する手段
CN111601605A (zh) * 2018-01-11 2020-08-28 生物技术Rna制药有限公司 用于施用rna的制剂

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996013599A1 (en) * 1994-11-01 1996-05-09 Winfried Wels Nucleic acid transfer system
US6353055B1 (en) * 1994-11-18 2002-03-05 Supratek Pharma Inc. Polynucleotide compositions
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells
FR2793414B1 (fr) * 1999-05-10 2003-05-23 Centre Nat Rech Scient Conjugue acide nucleique-anticorps pour delivrer un acide nucleique etranger dans les cellules
DE10015906B4 (de) * 2000-03-30 2006-09-07 Ibfb Pharma Gmbh Gerichteter Gentransfer in Thy-1-positive Zellen
US6586524B2 (en) * 2001-07-19 2003-07-01 Expression Genetics, Inc. Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier
US20030198627A1 (en) * 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
JP2005508396A (ja) * 2001-11-02 2005-03-31 イントラディグム、コーポレイション 核酸送達ビヒクルのための治療方法
AU2003207708A1 (en) * 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
AU2003217009A1 (en) * 2002-03-07 2003-09-16 Biocompatibles Uk Limited Compositions of polymers
WO2004014933A1 (en) * 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
EP1575574A4 (en) * 2002-10-30 2007-11-07 Blood Res Center METHOD FOR TREATING AND PREVENTING APOPTOSIS-BASED DISEASES BY MEANS OF RNA INTERFERENCE AGENTS

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104349794A (zh) * 2012-06-08 2015-02-11 埃泽瑞斯公司 信使rna的肺递送
CN109562059A (zh) * 2016-07-15 2019-04-02 生物技术Rna制药有限公司 用于施用rna的制剂
AU2017297822B2 (en) * 2016-07-15 2022-12-22 BioNTech SE Formulation for administration of RNA

Also Published As

Publication number Publication date
EP1592412A1 (en) 2005-11-09
BRPI0407107A (pt) 2006-01-24
US20060135453A1 (en) 2006-06-22
JP2006517793A (ja) 2006-08-03
CA2514536A1 (en) 2004-08-12
WO2004066989A1 (en) 2004-08-12

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