WO2008108344A1 - Ｌｃａｔ欠損症の遺伝子治療用細胞並びにこれを産生するのに用いられる複製欠損性レトロウイルスベクター及びプラスミド - Google Patents

Ｌｃａｔ欠損症の遺伝子治療用細胞並びにこれを産生するのに用いられる複製欠損性レトロウイルスベクター及びプラスミド Download PDF

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Publication number
WO2008108344A1
WO2008108344A1 PCT/JP2008/053791 JP2008053791W WO2008108344A1 WO 2008108344 A1 WO2008108344 A1 WO 2008108344A1 JP 2008053791 W JP2008053791 W JP 2008053791W WO 2008108344 A1 WO2008108344 A1 WO 2008108344A1
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Prior art keywords
cell
production
replication
plasmid
sequence
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PCT/JP2008/053791
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English (en)
French (fr)
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Atsuo Waki
Masayuki Aso
Junichi Fukuchi
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Cellgentech, Inc.
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Priority to JP2009502578A priority Critical patent/JPWO2008108344A1/ja
Publication of WO2008108344A1 publication Critical patent/WO2008108344A1/ja

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01026—Sterol O-acyltransferase (2.3.1.26)
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

　本発明の課題は、外来遺伝子としてのＬＣＡＴコードヌクレオチドを効率よく標的細胞で発現させることができる、患者に投与される遺伝子治療用細胞を産生するためのＬＣＡＴコードヌクレオチドを保持する複製欠損性レトロウイルスベクター、及び当該レトロウイルスベクターを産生するのに用いられるプラスミドを提供することにある。その解決手段としての本発明の複製欠損性レトロウイルスベクター産生用プラスミドは、レトロウイルス由来の５’側のＬＴＲ配列と３’側のＬＴＲ配列の間に、外来遺伝子としてＬＣＡＴコードヌクレオチド配列が挿入され、かつ、前記ＬＣＡＴコードヌクレオチド配列の５’側にコザックのコンセンサス配列及び前記レトロウイルスのパッケージングシグナル配列が連結されていることを特徴とする。
PCT/JP2008/053791 2007-03-02 2008-03-03 Ｌｃａｔ欠損症の遺伝子治療用細胞並びにこれを産生するのに用いられる複製欠損性レトロウイルスベクター及びプラスミド WO2008108344A1 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2009502578A JPWO2008108344A1 (ja) 2007-03-02 2008-03-03 Ｌｃａｔ欠損症の遺伝子治療用細胞並びに遺伝子治療用細胞組成物

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2007-052291 2007-03-02
JP2007052291 2007-03-02

Publications (1)

Publication Number Publication Date
WO2008108344A1 true WO2008108344A1 (ja) 2008-09-12

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PCT/JP2008/053791 WO2008108344A1 (ja) 2007-03-02 2008-03-03 Ｌｃａｔ欠損症の遺伝子治療用細胞並びにこれを産生するのに用いられる複製欠損性レトロウイルスベクター及びプラスミド

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JP (2) JPWO2008108344A1 (ja)
WO (1) WO2008108344A1 (ja)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011090091A1 (ja) * 2010-01-21 2011-07-28 セルジェンテック株式会社 遺伝子治療用脂肪細胞の調製のための外来遺伝子の導入に適した細胞集団かどうかを判定する方法
US8071085B2 (en) 2002-06-18 2011-12-06 Eisai Co., Ltd. Primary cultured adipocytes for gene therapy
JP2012530070A (ja) * 2009-06-12 2012-11-29 アルファコア ファーマ リミテッド ライアビリティ カンパニー 貧血および赤血球機能不全を治療するためのｌｃａｔの使用

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3122884B1 (en) * 2014-03-26 2019-12-25 Tocagen Inc. A retroviral vector having immune-stimulating activity

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH11511651A (ja) * 1995-05-10 1999-10-12 イントロヘーネ ベスローテン フェンノートシャップ 遺伝子治療に特に適した改良型レトロウイルスベクター
WO2003106663A1 (ja) * 2002-06-18 2003-12-24 エーザイ株式会社 遺伝子治療用初代培養脂肪細胞
JP2004516830A (ja) * 2000-12-05 2004-06-10 アプライド・リサーチ・システムズ・エイアールエス・ホールディング・ナムローゼ・フェンノートシャップ 哺乳動物系における組換えタンパク質の均質性および分泌の改良

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2731710B1 (fr) * 1995-03-14 1997-04-30 Rhone Poulenc Rorer Sa Virus recombinants exprimant la lecithine cholesterol acyltransferase et utilisations en therapie genique
JP4860612B2 (ja) * 2005-06-15 2012-01-25 タカラバイオ株式会社 脂肪細胞あるいは前駆脂肪細胞への遺伝子導入方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH11511651A (ja) * 1995-05-10 1999-10-12 イントロヘーネ ベスローテン フェンノートシャップ 遺伝子治療に特に適した改良型レトロウイルスベクター
JP2004516830A (ja) * 2000-12-05 2004-06-10 アプライド・リサーチ・システムズ・エイアールエス・ホールディング・ナムローゼ・フェンノートシャップ 哺乳動物系における組換えタンパク質の均質性および分泌の改良
WO2003106663A1 (ja) * 2002-06-18 2003-12-24 エーザイ株式会社 遺伝子治療用初代培養脂肪細胞

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8071085B2 (en) 2002-06-18 2011-12-06 Eisai Co., Ltd. Primary cultured adipocytes for gene therapy
JP2012530070A (ja) * 2009-06-12 2012-11-29 アルファコア ファーマ リミテッド ライアビリティ カンパニー 貧血および赤血球機能不全を治療するためのｌｃａｔの使用
WO2011090091A1 (ja) * 2010-01-21 2011-07-28 セルジェンテック株式会社 遺伝子治療用脂肪細胞の調製のための外来遺伝子の導入に適した細胞集団かどうかを判定する方法
JP5806791B2 (ja) * 2010-01-21 2015-11-10 セルジェンテック株式会社 遺伝子治療用脂肪細胞の調製のための外来遺伝子の導入に適した細胞集団かどうかを判定する方法

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JPWO2008108344A1 (ja) 2010-06-17

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