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AU2001268590A1 - Liver-specific gene expression cassettes, and methods of use - Google Patents

Liver-specific gene expression cassettes, and methods of use

Info

Publication number
AU2001268590A1
AU2001268590A1 AU2001268590A AU6859001A AU2001268590A1 AU 2001268590 A1 AU2001268590 A1 AU 2001268590A1 AU 2001268590 A AU2001268590 A AU 2001268590A AU 6859001 A AU6859001 A AU 6859001A AU 2001268590 A1 AU2001268590 A1 AU 2001268590A1
Authority
AU
Australia
Prior art keywords
liver
methods
gene expression
expression cassettes
specific gene
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.)
Abandoned
Application number
AU2001268590A
Other languages
English (en)
Inventor
Mark A Kay
Carol H. Miao
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.)
University of Washington
Leland Stanford Junior University
Original Assignee
University of Washington
Leland Stanford Junior University
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 University of Washington, Leland Stanford Junior University filed Critical University of Washington
Publication of AU2001268590A1 publication Critical patent/AU2001268590A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/108—Plasmid DNA episomal 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
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
AU2001268590A 2000-06-20 2001-06-19 Liver-specific gene expression cassettes, and methods of use Abandoned AU2001268590A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US21290200P 2000-06-20 2000-06-20
US60212902 2000-06-20
PCT/US2001/019634 WO2001098482A2 (fr) 2000-06-20 2001-06-19 Cassettes d'expression genetique specifiques du foie et methodes d'utilisation

Publications (1)

Publication Number Publication Date
AU2001268590A1 true AU2001268590A1 (en) 2002-01-02

Family

ID=22792849

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001268590A Abandoned AU2001268590A1 (en) 2000-06-20 2001-06-19 Liver-specific gene expression cassettes, and methods of use

Country Status (5)

Country Link
US (1) US7351813B2 (fr)
EP (1) EP1297120A2 (fr)
JP (1) JP2004500880A (fr)
AU (1) AU2001268590A1 (fr)
WO (1) WO2001098482A2 (fr)

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* 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
WO2004016791A1 (fr) * 2002-08-01 2004-02-26 Evolva Ltd Procedes pour melanger de grands nombres de genes heterologues
US20090010900A1 (en) * 2004-07-07 2009-01-08 The University Of North Carlolina At Chapel Hill Embryonic Stem Cell Derivatives, and Methods of Making and Using the Same
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
CN100338220C (zh) * 2005-03-28 2007-09-19 中国人民解放军军事医学科学院野战输血研究所 一种肝脏高效表达调控序列及其应用
EP2007795B1 (fr) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Protéines de capsides aav
US8309698B2 (en) * 2008-02-14 2012-11-13 Mogam Biotechnology Research Institute Expression vector suitable for expression of a coding sequence for gene therapy
EP4154903A1 (fr) * 2008-04-22 2023-03-29 Vib Vzw Éléments régulateurs d'acides nucléiques spécifiques au foie et procédés et leur utilisation
EP2310501B1 (fr) 2008-07-23 2013-07-03 Boehringer Ingelheim Pharma GmbH & Co. KG Nouveaux éléments régulateurs
KR101063247B1 (ko) 2008-10-22 2011-09-08 한국식품연구원 간 조직 특이적 유전자 ｍｍｌ2
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2394667A1 (fr) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vecteurs et séquences pour le traitement de maladies
JP2013530699A (ja) 2010-06-15 2013-08-01 セルラー ダイナミクス インターナショナル， インコーポレイテッド 生物学的応答の調査のための既製の幹細胞モデルの概要
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
AU2014218807A1 (en) 2013-02-22 2015-09-03 Cellular Dynamics International, Inc. Hepatocyte production via forward programming by combined genetic and chemical engineering
CN103667346B (zh) * 2013-12-17 2016-01-20 扬州大学 一种pRBE-HCR-hAAT-hFIXml质粒及其构建和应用
WO2015164228A1 (fr) 2014-04-21 2015-10-29 Cellular Dynamics International, Inc. Production d'hépatocytes par programmation progressive par génie génétique et chimique combinés
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016146757A1 (fr) * 2015-03-17 2016-09-22 Vrije Universiteit Brussel Systèmes d'expression spécifiques du foie optimisés pour fviii et fix
KR102178322B1 (ko) * 2015-06-23 2020-11-13 더 칠드런스 호스피탈 오브 필라델피아 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도
AU2017248659B2 (en) 2016-04-15 2022-08-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
JP2021510539A (ja) * 2018-01-16 2021-04-30 シーエルエス セラピューティクス リミテッドＣＬＳ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ デオキシリボヌクレアーゼ（ＤＮａｓｅ）活性を有する酵素の肝臓発現による疾患の処置
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP2021512871A (ja) 2018-02-01 2021-05-20 ホモロジー・メディシンズ・インコーポレイテッド Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びそれらの使用方法
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) * 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
WO2020084162A1 (fr) 2018-10-26 2020-04-30 Vrije Universiteit Brussel Éléments régulateurs d'acide nucléique spécifiques du foie et procédés et utilisation de ceux-ci
GB201818816D0 (en) 2018-11-19 2019-01-02 Synpromics Ltd Regulatory nucleic acid sequences
GB201907493D0 (en) 2019-05-28 2019-07-10 Ospedale San Raffaele Agents and methods for treating viral infections
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
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
GB202018657D0 (en) 2020-11-26 2021-01-13 Ospedale San Raffaele Srl Agents and methods for increasing liver immune response
KR102536321B1 (ko) * 2020-12-09 2023-05-26 재단법인 아산사회복지재단 인체 유래 유전자 발현 유도 모듈에 기반한 간 특이 유전자 발현 시스템
EP4267198A4 (fr) * 2020-12-22 2024-12-18 Cytotheryx, Inc. Méthodes d'administration biliaire, et compositions et kits destinés à être utilisés dans ces méthodes
EP4247414A4 (fr) 2021-01-20 2024-07-10 Curogen Technology Co., Ltd. Protéine ssu72 ou polynucléotide pour le traitement et le diagnostic du cancer du foie
CN117716020A (zh) 2021-05-07 2024-03-15 安斯泰来再生医药协会 产生成熟肝细胞的方法

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US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
WO1998024479A1 (fr) * 1996-12-02 1998-06-11 Cell Genesys, Inc. Apport d'adn a des cellules du foie s'effectuant a travers un vecteur viral adeno-associe
US6610906B1 (en) * 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
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

Also Published As

Publication number Publication date
JP2004500880A (ja) 2004-01-15
WO2001098482A3 (fr) 2002-05-30
US7351813B2 (en) 2008-04-01
EP1297120A2 (fr) 2003-04-02
US20020076798A1 (en) 2002-06-20
WO2001098482A2 (fr) 2001-12-27

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