JP7529286B2 - 肝特異的核酸調節エレメント並びにその方法及び使用 - Google Patents

肝特異的核酸調節エレメント並びにその方法及び使用 Download PDF

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JP7529286B2
JP7529286B2 JP2021522959A JP2021522959A JP7529286B2 JP 7529286 B2 JP7529286 B2 JP 7529286B2 JP 2021522959 A JP2021522959 A JP 2021522959A JP 2021522959 A JP2021522959 A JP 2021522959A JP 7529286 B2 JP7529286 B2 JP 7529286B2
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nucleic acid
liver
expression cassette
regulatory element
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JPWO2020084162A5 (enExample
JP2022505877A (ja
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キム チュア，レイ
フアンデンドリス，ティエリー
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Vrije Universiteit Brussel VUB
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    • 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
    • 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
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2021522959A 2018-10-26 2019-10-28 肝特異的核酸調節エレメント並びにその方法及び使用 Active JP7529286B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18202986 2018-10-26
EP18202986.8 2018-10-26
PCT/EP2019/079358 WO2020084162A1 (en) 2018-10-26 2019-10-28 Liver-specific nucleic acid regulatory elements and methods and use thereof

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JP2022505877A JP2022505877A (ja) 2022-01-14
JPWO2020084162A5 JPWO2020084162A5 (enExample) 2022-11-08
JP7529286B2 true JP7529286B2 (ja) 2024-08-06

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US (1) US20210403948A1 (enExample)
EP (1) EP3870237A1 (enExample)
JP (1) JP7529286B2 (enExample)
CN (1) CN112912112B (enExample)
WO (1) WO2020084162A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210034013A (ko) 2018-07-16 2021-03-29 박스알타 인코퍼레이티드 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
TWI851647B (zh) * 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
CN110791502B (zh) * 2019-12-04 2022-07-08 陕西理工大学 人源rbp4启动子片段及其应用
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
CA3213123A1 (en) * 2021-03-25 2022-09-29 Robert L. Kruse Hydrodynamic gene delivery
JP2025534900A (ja) * 2022-10-08 2025-10-21 リンイ バイオテック カンパニー リミテッド 遺伝子発現を増強するためのコンストラクト
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004500880A (ja) 2000-06-20 2004-01-15 ザ、ボード、オブ、トラスティーズ、オブ、ザ、リーランド、スタンフォード、ジュニア、ユニバーシティ 肝臓特異的な遺伝子発現カセット、及びその使用法
WO2011005968A1 (en) 2009-07-08 2011-01-13 Ucl Business Plc Codon-optimized factor vi i i variants and synthetic liver-specific promoter
JP2011517955A (ja) 2008-04-22 2011-06-23 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
JP2016500519A (ja) 2012-10-26 2016-01-14 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
JP2018513678A (ja) 2015-03-17 2018-05-31 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070161031A1 (en) * 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
EP2097538A4 (en) * 2006-12-07 2011-11-30 Switchgear Genomics TRANSCRIPTION REAGULATION ELEMENTS OF BIOLOGICAL PATHS, TOOLS AND METHODS
CN102618536B (zh) * 2008-02-14 2013-10-16 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
CA2765709A1 (en) * 2009-06-17 2010-12-23 Maine Standards Company, Llc Method for measuring lipoprotein-specific apolipoproteins
US8815779B2 (en) * 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
WO2011051450A1 (en) 2009-10-29 2011-05-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
JP2016522674A (ja) * 2012-05-16 2016-08-04 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
WO2013186398A1 (en) * 2012-06-15 2013-12-19 Fondazione Telethon Tfeb for use in treating obesity or metabolic syndrome
KR101429696B1 (ko) * 2012-11-21 2014-08-13 국립암센터 안전성 및 항암활성이 증가된 재조합 아데노바이러스 및 이의 용도
CN115141836B (zh) * 2015-10-28 2024-08-27 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004500880A (ja) 2000-06-20 2004-01-15 ザ、ボード、オブ、トラスティーズ、オブ、ザ、リーランド、スタンフォード、ジュニア、ユニバーシティ 肝臓特異的な遺伝子発現カセット、及びその使用法
JP2011517955A (ja) 2008-04-22 2011-06-23 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
WO2011005968A1 (en) 2009-07-08 2011-01-13 Ucl Business Plc Codon-optimized factor vi i i variants and synthetic liver-specific promoter
JP2016500519A (ja) 2012-10-26 2016-01-14 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
JP2018513678A (ja) 2015-03-17 2018-05-31 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Molecular Therapy，2014年，Vol.22, No.9，pp.1605-1613

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EP3870237A1 (en) 2021-09-01
JP2022505877A (ja) 2022-01-14
US20210403948A1 (en) 2021-12-30
CN112912112B (zh) 2024-12-17
WO2020084162A1 (en) 2020-04-30
CN112912112A (zh) 2021-06-04

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