JP2006517793A - スクリーニングアッセイ - Google Patents

スクリーニングアッセイ Download PDF

Info

Publication number
JP2006517793A
JP2006517793A JP2006501691A JP2006501691A JP2006517793A JP 2006517793 A JP2006517793 A JP 2006517793A JP 2006501691 A JP2006501691 A JP 2006501691A JP 2006501691 A JP2006501691 A JP 2006501691A JP 2006517793 A JP2006517793 A JP 2006517793A
Authority
JP
Japan
Prior art keywords
gene
target gene
pei
ssrna
cell
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
JP2006501691A
Other languages
English (en)
Japanese (ja)
Other versions
JP2006517793A5 (https=
Inventor
ジャン−シャルル・ボローニャ
ジョナサン・ホール
フランソワ・ジャン−シャルル・ナット
ヤン・ヴァイラー
Original Assignee
ノバルティス アクチエンゲゼルシャフト
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 ノバルティス アクチエンゲゼルシャフト filed Critical ノバルティス アクチエンゲゼルシャフト
Publication of JP2006517793A publication Critical patent/JP2006517793A/ja
Publication of JP2006517793A5 publication Critical patent/JP2006517793A5/ja
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
    • C12N15/111—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
    • 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
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/50—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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • 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)
JP2006501691A 2003-01-31 2004-01-30 スクリーニングアッセイ Pending JP2006517793A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44397703P 2003-01-31 2003-01-31
PCT/EP2004/000897 WO2004066989A1 (en) 2003-01-31 2004-01-30 Down-regulation of target-gene with pei/single-stranded oligoribonucleotide comp lexes

Publications (2)

Publication Number Publication Date
JP2006517793A true JP2006517793A (ja) 2006-08-03
JP2006517793A5 JP2006517793A5 (https=) 2007-02-15

Family

ID=32825395

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2006501691A Pending JP2006517793A (ja) 2003-01-31 2004-01-30 スクリーニングアッセイ

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
JP2019527685A (ja) * 2016-07-15 2019-10-03 バイオンテック エールエヌアー ファーマシューティカルズ ゲーエムベーハー Ｒｎａの投与のための配合物
JP2023082121A (ja) * 2018-01-11 2023-06-13 バイオンテック・エスイー Ｒｎａの投与のための配合物

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
EA037448B1 (ru) * 2012-06-08 2021-03-30 Этрис Гмбх Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999006055A1 (en) * 1997-08-01 1999-02-11 Supratek Pharma Inc. Polynucleotide compositions
JP2002543810A (ja) * 1999-05-10 2002-12-24 サントル・ナシオナル・ドゥ・ラ・ルシェルシュ・シアンティフィク 外来性核酸を細胞に送達するための核酸−抗体複合体
JP2006517916A (ja) * 2002-10-30 2006-08-03 ザ シービーアール インスティテュート フォー バイオメディカル リサーチ インコーポレーティッド Ｒｎａ干渉物質を用いてアポトーシス関連疾患を治療および予防する方法

Family Cites Families (9)

* 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
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999006055A1 (en) * 1997-08-01 1999-02-11 Supratek Pharma Inc. Polynucleotide compositions
JP2002543810A (ja) * 1999-05-10 2002-12-24 サントル・ナシオナル・ドゥ・ラ・ルシェルシュ・シアンティフィク 外来性核酸を細胞に送達するための核酸−抗体複合体
JP2006517916A (ja) * 2002-10-30 2006-08-03 ザ シービーアール インスティテュート フォー バイオメディカル リサーチ インコーポレーティッド Ｒｎａ干渉物質を用いてアポトーシス関連疾患を治療および予防する方法

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019527685A (ja) * 2016-07-15 2019-10-03 バイオンテック エールエヌアー ファーマシューティカルズ ゲーエムベーハー Ｒｎａの投与のための配合物
US11318195B2 (en) 2016-07-15 2022-05-03 BioNTech SE Formulation for administration of RNA
JP7139307B2 (ja) 2016-07-15 2022-09-20 バイオンテック・エスイー Ｒｎａの投与のための配合物
JP2022174191A (ja) * 2016-07-15 2022-11-22 バイオンテック・エスイー Ｒｎａの投与のための配合物
JP7675052B2 (ja) 2016-07-15 2025-05-12 バイオンテック・エスイー Ｒｎａの投与のための配合物
JP2023082121A (ja) * 2018-01-11 2023-06-13 バイオンテック・エスイー Ｒｎａの投与のための配合物

Also Published As

Publication number Publication date
CN1744886A (zh) 2006-03-08
EP1592412A1 (en) 2005-11-09
BRPI0407107A (pt) 2006-01-24
US20060135453A1 (en) 2006-06-22
CA2514536A1 (en) 2004-08-12
WO2004066989A1 (en) 2004-08-12

Similar Documents

Publication Publication Date Title
JP5243789B2 (ja) 二本鎖ｒｎａによる遺伝子発現の特異的阻害のための方法及び組成物
US10233450B2 (en) Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
RU2399671C2 (ru) Способный к интерференции дуплекс рнк, имеющий "тупые" концы и 3'-модификации
JP6069096B2 (ja) より新規形態の干渉ｒｎａ分子
JP2006517793A (ja) スクリーニングアッセイ
CN101213300B (zh) 用于rna干扰的寡核苷酸及其生物学应用
Tang et al. Differential gene silencing induced by short interfering RNA in cultured pine cells associates with the cell cycle phase
US20060287269A1 (en) Short interfering nucleic acid hybrids and methods thereof
WO2005068630A1 (ja) 干渉用二重鎖ｒｎａ
Kim et al. Enhancing RNAi with Synthetic RNA Duplexes
HK1171046A (en) Interfering rna duplex having blunt-ends and 3'-modifications

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20060926

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20061220

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20061220

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20091215

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20100518