WO2003046186A1 - siRNA EXPRESSION SYSTEM AND METHOD FOR PRODUCING FUNCTIONAL GENE KNOCK-DOWN CELLS USING THE SYSTEM - Google Patents

siRNA EXPRESSION SYSTEM AND METHOD FOR PRODUCING FUNCTIONAL GENE KNOCK-DOWN CELLS USING THE SYSTEM Download PDF

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Publication number
WO2003046186A1
WO2003046186A1 PCT/JP2002/012447 JP0212447W WO03046186A1 WO 2003046186 A1 WO2003046186 A1 WO 2003046186A1 JP 0212447 W JP0212447 W JP 0212447W WO 03046186 A1 WO03046186 A1 WO 03046186A1
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WIPO (PCT)
Prior art keywords
sirna expression
rna
functional gene
gene knock
producing functional
Prior art date
Application number
PCT/JP2002/012447
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English (en)
French (fr)
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WO2003046186A8 (fr
Inventor
Kazunari Taira
Makoto Miyagishi
Original Assignee
Toudai Tlo, Ltd.
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
Priority claimed from PCT/JP2002/011293 external-priority patent/WO2003046173A1/ja
Priority to US10/470,197 priority Critical patent/US20050048647A1/en
Priority to JP2003547618A priority patent/JP3642573B2/ja
Priority to DE60237919T priority patent/DE60237919D1/de
Priority to AT02785973T priority patent/ATE483805T1/de
Priority to CA002468955A priority patent/CA2468955A1/en
Application filed by Toudai Tlo, Ltd. filed Critical Toudai Tlo, Ltd.
Priority to KR10-2004-7008244A priority patent/KR20040072643A/ko
Priority to EP02785973A priority patent/EP1462525B1/en
Priority to AU2002354121A priority patent/AU2002354121A1/en
Publication of WO2003046186A1 publication Critical patent/WO2003046186A1/ja
Publication of WO2003046186A8 publication Critical patent/WO2003046186A8/ja
Priority to HK05109992.4A priority patent/HK1078105A1/xx

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    • 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
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • 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
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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 N.A.
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/12—Applications; Uses in screening processes in functional genomics, i.e. for the determination of gene function

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
PCT/JP2002/012447 2001-11-28 2002-11-28 siRNA EXPRESSION SYSTEM AND METHOD FOR PRODUCING FUNCTIONAL GENE KNOCK-DOWN CELLS USING THE SYSTEM WO2003046186A1 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
AU2002354121A AU2002354121A1 (en) 2001-11-28 2002-11-28 siRNA Expression System and Method for Producing Functional Gene Knockdown Cell Using the Same
JP2003547618A JP3642573B2 (ja) 2001-11-28 2002-11-28 ｓｉＲＮＡ発現システムおよびこれを用いた機能遺伝子ノックダウン細胞等の生産方法
DE60237919T DE60237919D1 (de) 2001-11-28 2002-11-28 siRNA-EXPRESSIONSSYSTEM UND VERFAHREN ZUR HERSTELLUNG EINER ZELLE O. Ä. MIT KNOCKDOWN EINES FUNKTIONELLEN GENS UNTER VERWENDUNG DESSELBEN
AT02785973T ATE483805T1 (de) 2001-11-28 2002-11-28 Sirna-expressionssystem und verfahren zur herstellung einer zelle o. ä. mit knockdown eines funktionellen gens unter verwendung desselben
CA002468955A CA2468955A1 (en) 2001-11-28 2002-11-28 Sirna expression system and process for producing functional gene knockdown cell or the like using the same
US10/470,197 US20050048647A1 (en) 2001-11-28 2002-11-28 Sirna expression system and process for producing functional gene knockdown cell or the like using the same
KR10-2004-7008244A KR20040072643A (ko) 2001-11-28 2002-11-28 ｓｉＲＮＡ 발현시스템 및 이것을 이용한 기능유전자넉다운 세포 등의 생산방법
EP02785973A EP1462525B1 (en) 2001-11-28 2002-11-28 siRNA EXPRESSION SYSTEM AND PROCESS FOR PRODUCING FUNCTIONAL GENE KNOCKDOWN CELL OR THE LIKE USING THE SAME
HK05109992.4A HK1078105A1 (en) 2001-11-28 2005-11-09 Sirna expression system and process for producing a cell which functional gene has been knocked down or the like using the same sirna

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
JP2001-363385 2001-11-28
JP2001363385 2001-11-28
JPPCT/JP02-11293 2002-10-30
PCT/JP2002/011293 WO2003046173A1 (fr) 2001-11-28 2002-10-30 Systeme d'expression d'arn si et procede de production de cellules d'inactivation de genes fonctionnelles et analogues au moyen de ce systeme

Publications (2)

Publication Number Publication Date
WO2003046186A1 true WO2003046186A1 (en) 2003-06-05
WO2003046186A8 WO2003046186A8 (fr) 2005-09-29

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Family Applications (1)

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PCT/JP2002/012447 WO2003046186A1 (en) 2001-11-28 2002-11-28 siRNA EXPRESSION SYSTEM AND METHOD FOR PRODUCING FUNCTIONAL GENE KNOCK-DOWN CELLS USING THE SYSTEM

Country Status (5)

Country Link
EP (1) EP1462525B1 (ja)
JP (1) JP3642573B2 (ja)
AU (1) AU2002354121A1 (ja)
CA (1) CA2468955A1 (ja)
WO (1) WO2003046186A1 (ja)

Cited By (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003091433A1 (fr) * 2002-04-26 2003-11-06 National Institute Of Advanced Industrial Science And Technology Systeme d'expression pour molecule d'arn a tige-boucle ayant un effet d'arni
WO2004022777A1 (en) * 2002-09-04 2004-03-18 Johnson & Johnson Research Pty Ltd METHODS USING dsDNA TO MEDIATE RNA INTERFERENCE (RNAi)
WO2005035778A1 (ja) * 2003-10-09 2005-04-21 Kyowa Hakko Kogyo Co., Ltd. α1,6-フコシルトランスフェラーゼの機能を抑制するRNAを用いた抗体組成物の製造法
WO2005056792A1 (ja) * 2003-12-12 2005-06-23 National Institute Of Advanced Industrial Science And Technology インターフェロン応答が軽減された長い干渉用二重鎖ｒｎａ
EP1545624A2 (en) * 2001-12-27 2005-06-29 ALLELE BIOTECHNOLOGY & PHARMACEUTICALS, INC. Compositions for dna mediated gene silencing
WO2005068630A1 (ja) * 2003-12-16 2005-07-28 National Institute Of Advanced Industrial Science And Technology 干渉用二重鎖ｒｎａ
JP2005533484A (ja) * 2002-01-22 2005-11-10 コールド スプリング ハーバー ラボラトリー Ｒｎａ干渉のための方法及び組成物
EP1613722A4 (en) * 2002-10-18 2006-08-02 Genta Salus Llc OLIGONUCLEOTIDES INHIBITORS BASED ON RNA
JP2007515966A (ja) * 2003-12-23 2007-06-21 ザ・トラスティーズ・オブ・ザ・ユニバーシティ・オブ・ペンシルバニア 疾患の併用療法のための組成物および方法
JP2007527244A (ja) * 2004-03-05 2007-09-27 ベニテック インコーポレイテッド ＲＮＡｉ作用媒介物の同時デリバリーのためのマルチプロモーター発現カセット
JPWO2005017154A1 (ja) * 2003-08-18 2007-11-01 財団法人ヒューマンサイエンス振興財団 改良されたｓｉＲＮＡ分子およびこれを用いた遺伝子発現の抑制法
WO2010071206A1 (ja) * 2008-12-18 2010-06-24 国立大学法人名古屋大学 ｓｉＲＮＡ転写／発現用ＤＮＡコンストラクト及びその利用
US7893036B2 (en) 2001-07-12 2011-02-22 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US10233247B2 (en) 1999-04-09 2019-03-19 Kyowa Hakko Kirin Co., Ltd Method of modulating the activity of functional immune molecules
US10233475B2 (en) 2000-10-06 2019-03-19 Kyowa Hakko Kirin Co., Ltd Antibody composition-producing cell

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US8202846B2 (en) 2000-03-16 2012-06-19 Cold Spring Harbor Laboratory Methods and compositions for RNA interference
CA2403397A1 (en) 2000-03-16 2001-09-20 Genetica, Inc. Methods and compositions for rna interference
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
ES2312753T5 (es) 2002-02-14 2012-12-13 City Of Hope Procedimientos para producir moléculas de ARN de interferencia en células de mamífero y usos terapéuticos para tales moléculas
JP2005517450A (ja) * 2002-02-20 2005-06-16 サーナ・セラピューティクス・インコーポレイテッド 短干渉核酸（ｓｉＮＡ）を用いるＲＮＡ干渉媒介性標的発見および標的評価
US20050059044A1 (en) * 2003-06-03 2005-03-17 Graham Michael Wayne Double-stranded nucleic acid
US20080207539A1 (en) * 2003-09-01 2008-08-28 Patrick Arbuthnot Self-Processing Rna Expression Cassette
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
FR2877674A1 (fr) * 2004-11-08 2006-05-12 Genoxay Sa Animaux modeles comprenant au moins un transgene et une sequence permettant l'expression controlee d'un rna interferant avec ledit transgene
EP1681347A1 (en) * 2005-01-18 2006-07-19 Metanomics GmbH & Co. KGaA Improved methods for double-stranded RNA mediated gene silencing
WO2006116756A1 (en) * 2005-04-28 2006-11-02 Benitec, Limited. Multiple-rnai expression cassettes for simultaneous delivery of rnai agents related to heterozygotic expression patterns
US20100299771A1 (en) * 2007-09-17 2010-11-25 Helmholtz Zentrum Munchen- Deutsches Forschungzentrum Fur Gesundheit Und Umwelt(Gmbh) MEANS AND METHODS FOR shRNA MEDIATED CONDITIONAL KNOCKDOWN OF GENES

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Cited By (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10233247B2 (en) 1999-04-09 2019-03-19 Kyowa Hakko Kirin Co., Ltd Method of modulating the activity of functional immune molecules
US10233475B2 (en) 2000-10-06 2019-03-19 Kyowa Hakko Kirin Co., Ltd Antibody composition-producing cell
US9850487B2 (en) 2001-07-12 2017-12-26 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US8232260B2 (en) 2001-07-12 2012-07-31 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US10731155B2 (en) 2001-07-12 2020-08-04 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US7893036B2 (en) 2001-07-12 2011-02-22 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
US8530438B2 (en) 2001-07-12 2013-09-10 University Of Massachusetts Vivo production of small interfering RNAs that mediate gene silencing
US8557785B2 (en) 2001-07-12 2013-10-15 University Of Massachusetts In vivo production of small interfering RNAS that mediate gene silencing
US9175287B2 (en) 2001-07-12 2015-11-03 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
EP1545624A2 (en) * 2001-12-27 2005-06-29 ALLELE BIOTECHNOLOGY & PHARMACEUTICALS, INC. Compositions for dna mediated gene silencing
EP1545624A4 (en) * 2001-12-27 2006-03-29 Allele Biotechnology & Pharmac COMPOSITIONS FOR DNA-MEDIATED GEN SILENCING
US7294504B1 (en) 2001-12-27 2007-11-13 Allele Biotechnology & Pharmaceuticals, Inc. Methods and compositions for DNA mediated gene silencing
US7422896B1 (en) 2001-12-27 2008-09-09 Allele Biotechnology & Pharmaceuticals, Inc. Compositions for DNA mediated gene silencing
US7625750B2 (en) 2001-12-27 2009-12-01 Allele Biotechnology & Pharmaceuticals, Inc. Compositions for DNA mediated gene silencing
JP2005533484A (ja) * 2002-01-22 2005-11-10 コールド スプリング ハーバー ラボラトリー Ｒｎａ干渉のための方法及び組成物
JP4758067B2 (ja) * 2002-01-22 2011-08-24 コールド スプリング ハーバー ラボラトリー Ｒｎａ干渉のための方法及び組成物
JP2011135882A (ja) * 2002-01-22 2011-07-14 Cold Spring Harbor Lab Ｒｎａ干渉のための方法及び組成物
WO2003091433A1 (fr) * 2002-04-26 2003-11-06 National Institute Of Advanced Industrial Science And Technology Systeme d'expression pour molecule d'arn a tige-boucle ayant un effet d'arni
WO2004022777A1 (en) * 2002-09-04 2004-03-18 Johnson & Johnson Research Pty Ltd METHODS USING dsDNA TO MEDIATE RNA INTERFERENCE (RNAi)
EP1613722A4 (en) * 2002-10-18 2006-08-02 Genta Salus Llc OLIGONUCLEOTIDES INHIBITORS BASED ON RNA
JP4572299B2 (ja) * 2003-08-18 2010-11-04 財団法人ヒューマンサイエンス振興財団 改良されたｓｉＲＮＡ分子およびこれを用いた遺伝子発現の抑制法
JPWO2005017154A1 (ja) * 2003-08-18 2007-11-01 財団法人ヒューマンサイエンス振興財団 改良されたｓｉＲＮＡ分子およびこれを用いた遺伝子発現の抑制法
CN1867672B (zh) * 2003-08-18 2012-11-14 财团法人日本健康科学振兴财团 改良的siRNA分子和应用该改良的siRNA分子的抑制基因表达的方法
WO2005035778A1 (ja) * 2003-10-09 2005-04-21 Kyowa Hakko Kogyo Co., Ltd. α1,6-フコシルトランスフェラーゼの機能を抑制するRNAを用いた抗体組成物の製造法
JPWO2005035778A1 (ja) * 2003-10-09 2006-12-21 協和醗酵工業株式会社 α１，６−フコシルトランスフェラーゼの機能を抑制するＲＮＡを用いた抗体組成物の製造法
WO2005056792A1 (ja) * 2003-12-12 2005-06-23 National Institute Of Advanced Industrial Science And Technology インターフェロン応答が軽減された長い干渉用二重鎖ｒｎａ
JP2011087601A (ja) * 2003-12-16 2011-05-06 National Institute Of Advanced Industrial Science & Technology 干渉用二重鎖ｒｎａ
WO2005068630A1 (ja) * 2003-12-16 2005-07-28 National Institute Of Advanced Industrial Science And Technology 干渉用二重鎖ｒｎａ
JPWO2005068630A1 (ja) * 2003-12-16 2007-07-26 独立行政法人産業技術総合研究所 干渉用二重鎖ｒｎａ
JP2007515966A (ja) * 2003-12-23 2007-06-21 ザ・トラスティーズ・オブ・ザ・ユニバーシティ・オブ・ペンシルバニア 疾患の併用療法のための組成物および方法
JP2007527244A (ja) * 2004-03-05 2007-09-27 ベニテック インコーポレイテッド ＲＮＡｉ作用媒介物の同時デリバリーのためのマルチプロモーター発現カセット
JP4763681B2 (ja) * 2004-03-05 2011-08-31 ベニテック インコーポレイテッド ＲＮＡｉ作用媒介物の同時デリバリーのためのマルチプロモーター発現カセット
JPWO2010071206A1 (ja) * 2008-12-18 2012-05-31 国立大学法人名古屋大学 ｓｉＲＮＡ転写／発現用ＤＮＡコンストラクト及びその利用
WO2010071206A1 (ja) * 2008-12-18 2010-06-24 国立大学法人名古屋大学 ｓｉＲＮＡ転写／発現用ＤＮＡコンストラクト及びその利用

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JP3642573B2 (ja) 2005-04-27
WO2003046186A8 (fr) 2005-09-29
JPWO2003046186A1 (ja) 2005-04-07
CA2468955A1 (en) 2003-06-05
AU2002354121A1 (en) 2003-06-10
EP1462525B1 (en) 2010-10-06
EP1462525A1 (en) 2004-09-29

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