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WO2008133137A1 - 遺伝子治療のためのベクター - Google Patents

遺伝子治療のためのベクター Download PDF

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Publication number
WO2008133137A1
WO2008133137A1 PCT/JP2008/057414 JP2008057414W WO2008133137A1 WO 2008133137 A1 WO2008133137 A1 WO 2008133137A1 JP 2008057414 W JP2008057414 W JP 2008057414W WO 2008133137 A1 WO2008133137 A1 WO 2008133137A1
Authority
WO
WIPO (PCT)
Prior art keywords
transcription
unit
vector
retroviral vector
manner
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.)
Ceased
Application number
PCT/JP2008/057414
Other languages
English (en)
French (fr)
Inventor
Hideto Chono
Kazuya Matsumoto
Hajime Matsumura
Junichi Mineno
Ikunoshin Kato
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.)
Takara Bio Inc
Original Assignee
Takara Bio Inc
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 Takara Bio Inc filed Critical Takara Bio Inc
Priority to CN200880012249.7A priority Critical patent/CN101663397B/zh
Priority to JP2009511821A priority patent/JP5484897B2/ja
Priority to KR1020097021664A priority patent/KR101252065B1/ko
Priority to HK10108182.9A priority patent/HK1141831B/xx
Priority to US12/596,744 priority patent/US20100137415A1/en
Priority to EP08740493A priority patent/EP2138580B1/en
Publication of WO2008133137A1 publication Critical patent/WO2008133137A1/ja
Anticipated expiration legal-status Critical
Priority to US14/169,361 priority patent/US20140170709A1/en
Ceased legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/867Retroviral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • AIDS & HIV (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

 本発明は、転写調節配列と、前記配列により発現の制御が可能な形態に配置された一本鎖RNA特異的なエンドリボヌクレア-ゼ活性を有するポリペプチドをコードする遺伝子からなる転写ユニットを含有するレトロウイルスベクターであって、前記ユニットからのmRNAの転写方向がレトロウイルスベクターのRNAゲノムの転写方向に対して逆向きとなるように前記ユニットが配置されていることを特徴とするレトロウイルスベクターを提供する。前記の構成のベクターを使用することにより、高い遺伝子導入効率を示すウイルス上清を調製することができる。本発明のレトロウイルスベクターは、がんやウイルス感染症の治療、予防に有用である。
PCT/JP2008/057414 2007-04-20 2008-04-16 遺伝子治療のためのベクター Ceased WO2008133137A1 (ja)

Priority Applications (7)

Application Number Priority Date Filing Date Title
CN200880012249.7A CN101663397B (zh) 2007-04-20 2008-04-16 用于基因治疗的载体
JP2009511821A JP5484897B2 (ja) 2007-04-20 2008-04-16 遺伝子治療のためのベクター
KR1020097021664A KR101252065B1 (ko) 2007-04-20 2008-04-16 유전자 치료를 위한 벡터
HK10108182.9A HK1141831B (en) 2007-04-20 2008-04-16 Vector for gene therapy
US12/596,744 US20100137415A1 (en) 2007-04-20 2008-04-16 Vector for gene therapy
EP08740493A EP2138580B1 (en) 2007-04-20 2008-04-16 Vector for gene therapy
US14/169,361 US20140170709A1 (en) 2007-04-20 2014-01-31 Vector for gene therapy

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
JP2007-112178 2007-04-20
JP2007112178 2007-04-20
JP2007-138889 2007-05-25
JP2007138889 2007-05-25
JP2007-268310 2007-10-15
JP2007268310 2007-10-15
JP2008025248 2008-02-05
JP2008-025248 2008-02-05

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US12/596,744 A-371-Of-International US20100137415A1 (en) 2007-04-20 2008-04-16 Vector for gene therapy
US14/169,361 Division US20140170709A1 (en) 2007-04-20 2014-01-31 Vector for gene therapy

Publications (1)

Publication Number Publication Date
WO2008133137A1 true WO2008133137A1 (ja) 2008-11-06

Family

ID=39925593

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/JP2008/057414 Ceased WO2008133137A1 (ja) 2007-04-20 2008-04-16 遺伝子治療のためのベクター

Country Status (7)

Country Link
US (2) US20100137415A1 (ja)
EP (1) EP2138580B1 (ja)
JP (1) JP5484897B2 (ja)
KR (1) KR101252065B1 (ja)
CN (1) CN101663397B (ja)
TW (1) TWI411682B (ja)
WO (1) WO2008133137A1 (ja)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012046727A1 (ja) 2010-10-05 2012-04-12 タカラバイオ株式会社 ウイルスベクターの製造方法
WO2012118092A1 (ja) * 2011-03-01 2012-09-07 タカラバイオ株式会社 融合タンパク質
WO2013141133A1 (ja) 2012-03-22 2013-09-26 タカラバイオ株式会社 ウイルスベクターの製造方法
WO2014092090A1 (ja) * 2012-12-11 2014-06-19 タカラバイオ株式会社 ウイルスベクター産生細胞及びベクターの製造方法
JP2020078326A (ja) * 2009-03-02 2020-05-28 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 腫瘍選択的e1aおよびe1b変異体

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1921136B1 (en) 2005-08-16 2012-12-12 Takara Bio, Inc. Nucleic acid for treatment or prevention of immunodeficiency virus infection
WO2019083976A1 (en) 2017-10-23 2019-05-02 Yuntao Wu REV-DEPENDENT LENTIVIRAL VACCINE PARTICLES FOR REDUCING VIRAL REBOUND AND IN VIVO VIRAL TANKS
FR3158515A1 (fr) * 2024-01-23 2025-07-25 Asfalia Biologics Production de vecteurs toxiques
FR3158516A1 (fr) * 2024-01-23 2025-07-25 Asfalia Biologics Production de vecteurs toxiques
FR3158514A1 (fr) * 2024-01-23 2025-07-25 Asfalia Biologics Système de régulation de l’expression d’un transgène d’intérêt

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Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020078326A (ja) * 2009-03-02 2020-05-28 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 腫瘍選択的e1aおよびe1b変異体
US11618888B2 (en) 2009-03-02 2023-04-04 The Regents Of The University Of California Tumor-selective E1A and E1B mutants
US12195766B2 (en) 2009-03-02 2025-01-14 The Regents Of The University Of California Tumor-selective E1a and E1b mutants
WO2012046727A1 (ja) 2010-10-05 2012-04-12 タカラバイオ株式会社 ウイルスベクターの製造方法
US9102943B2 (en) 2010-10-05 2015-08-11 Takara Bio Inc. Method for producing virus vector
WO2012118092A1 (ja) * 2011-03-01 2012-09-07 タカラバイオ株式会社 融合タンパク質
WO2013141133A1 (ja) 2012-03-22 2013-09-26 タカラバイオ株式会社 ウイルスベクターの製造方法
US9399780B2 (en) 2012-03-22 2016-07-26 Takara Bio Inc. Method for producing viral vector
WO2014092090A1 (ja) * 2012-12-11 2014-06-19 タカラバイオ株式会社 ウイルスベクター産生細胞及びベクターの製造方法

Also Published As

Publication number Publication date
JPWO2008133137A1 (ja) 2010-07-22
HK1141831A1 (en) 2010-11-19
TWI411682B (zh) 2013-10-11
KR101252065B1 (ko) 2013-04-12
CN101663397A (zh) 2010-03-03
EP2138580A1 (en) 2009-12-30
EP2138580A4 (en) 2010-12-29
EP2138580B1 (en) 2013-03-20
US20100137415A1 (en) 2010-06-03
KR20100015650A (ko) 2010-02-12
CN101663397B (zh) 2015-04-22
US20140170709A1 (en) 2014-06-19
TW200902715A (en) 2009-01-16
JP5484897B2 (ja) 2014-05-07

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