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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
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 (en) 2000-06-20 2001-06-19 Liver-specific gene expression cassettes, and methods of use

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 (en)
EP (1) EP1297120A2 (en)
JP (1) JP2004500880A (en)
AU (1) AU2001268590A1 (en)
WO (1) WO2001098482A2 (en)

Families Citing this family (37)

* 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 (en) * 2002-08-01 2004-02-26 Evolva Ltd Methods of mixing large numbers of heterologous genes
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 (en) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Improved expression of Factor IX in gene therapy vectors
CN100338220C (en) * 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 Liver highly expression regulating gene sequence and its application
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
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 (en) * 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
EP2310501B1 (en) 2008-07-23 2013-07-03 Boehringer Ingelheim Pharma GmbH & Co. KG Novel regulatory elements
KR101063247B1 (en) 2008-10-22 2011-09-08 한국식품연구원 Liver tissue specific gene MML2
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
JP2013530699A (en) 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 Overview of ready-made stem cell models for investigating biological responses
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 (en) * 2013-12-17 2016-01-20 扬州大学 A kind of pRBE-HCR-hAAT-hFIXml plasmid and structure thereof and application
WO2015164228A1 (en) 2014-04-21 2015-10-29 Cellular Dynamics International, Inc. Hepatocyte production via forward programming by combined genetic and chemical engineering
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016146757A1 (en) * 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
KR102178322B1 (en) * 2015-06-23 2020-11-13 더 칠드런스 호스피탈 오브 필라델피아 Modified Factor IX, and compositions, methods and uses for delivering genes to cells, organs and tissues
AU2017248659B2 (en) 2016-04-15 2022-08-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
JP2021510539A (en) * 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 Treatment of diseases by liver expression of enzymes with deoxyribonuclease (DNase) activity
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 (en) 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 Adeno-associated virus compositions for restoring PAH gene function and how to use them
JP7244547B2 (en) 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 Adeno-associated virus compositions and methods of use thereof for restoring F8 gene function
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 (en) 2018-10-26 2020-04-30 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods and use thereof
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 (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
TW202208632A (en) 2020-05-27 2022-03-01 美商同源醫藥公司 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
GB202018657D0 (en) 2020-11-26 2021-01-13 Ospedale San Raffaele Srl Agents and methods for increasing liver immune response
KR102536321B1 (en) * 2020-12-09 2023-05-26 재단법인 아산사회복지재단 Liver-specific gene expression cassette based on human-derived gene expression module
EP4267198A4 (en) * 2020-12-22 2024-12-18 Cytotheryx, Inc. Biliary delivery methods, compositions and kits for use therein
EP4247414A4 (en) 2021-01-20 2024-07-10 Curogen Technology Co., Ltd. Ssu72 protein or polynucleotide for treating and diagnosing liver cancer
CN117716020A (en) 2021-05-07 2024-03-15 安斯泰来再生医药协会 Method for producing mature hepatocytes

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US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
WO1998024479A1 (en) * 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
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 (en) 2004-01-15
WO2001098482A3 (en) 2002-05-30
US7351813B2 (en) 2008-04-01
EP1297120A2 (en) 2003-04-02
US20020076798A1 (en) 2002-06-20
WO2001098482A2 (en) 2001-12-27

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