CN112912112B - 肝特异性核酸调节元件以及其方法及用途 - Google Patents

肝特异性核酸调节元件以及其方法及用途 Download PDF

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CN112912112B
CN112912112B CN201980070613.3A CN201980070613A CN112912112B CN 112912112 B CN112912112 B CN 112912112B CN 201980070613 A CN201980070613 A CN 201980070613A CN 112912112 B CN112912112 B CN 112912112B
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seq
nucleic acid
liver
expression cassette
sequence
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CN112912112A (zh
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L.K.蔡
T.范登德里斯奇
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Vrije Universiteit Brussel VUB
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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
    • C12N15/63—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)
  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • Molecular Biology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Virology (AREA)
  • Physics & Mathematics (AREA)
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  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (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)
CN201980070613.3A 2018-10-26 2019-10-28 肝特异性核酸调节元件以及其方法及用途 Active CN112912112B (zh)

Applications Claiming Priority (3)

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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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CN112912112A CN112912112A (zh) 2021-06-04
CN112912112B true CN112912112B (zh) 2024-12-17

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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
CA3106590A1 (en) 2018-07-16 2020-01-23 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
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
WO2022204535A1 (en) * 2021-03-25 2022-09-29 The Johns Hopkins University Hydrodynamic gene delivery
EP4598590A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102618536A (zh) * 2008-02-14 2012-08-01 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

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
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
WO2008073303A2 (en) * 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
WO2009130208A1 (en) 2008-04-22 2009-10-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
JP2012530900A (ja) * 2009-06-17 2012-12-06 メイン スタンダーズ カンパニー リミテッド ライアビリティ カンパニー リポタンパク質特異的アポリポタンパク質を測定する方法
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US8815779B2 (en) * 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
CA2778945A1 (en) 2009-10-29 2011-05-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
EP2850184A4 (en) * 2012-05-16 2016-01-27 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION
WO2013186398A1 (en) * 2012-06-15 2013-12-19 Fondazione Telethon Tfeb for use in treating obesity or metabolic syndrome
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
KR101429696B1 (ko) * 2012-11-21 2014-08-13 국립암센터 안전성 및 항암활성이 증가된 재조합 아데노바이러스 및 이의 용도
EP3270944B1 (en) * 2015-03-17 2019-10-23 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
AU2016343887B2 (en) * 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102618536A (zh) * 2008-02-14 2012-08-01 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

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JP2022505877A (ja) 2022-01-14
EP3870237A1 (en) 2021-09-01
US20210403948A1 (en) 2021-12-30
JP7529286B2 (ja) 2024-08-06
WO2020084162A1 (en) 2020-04-30
CN112912112A (zh) 2021-06-04

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