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WO1999003981A8 - Nucleic acid expression constructs incorporating a heterologous intron whose natural position is within the 5'untranslated region of its gene - Google Patents

Nucleic acid expression constructs incorporating a heterologous intron whose natural position is within the 5'untranslated region of its gene

Info

Publication number
WO1999003981A8
WO1999003981A8 PCT/GB1998/002130 GB9802130W WO9903981A8 WO 1999003981 A8 WO1999003981 A8 WO 1999003981A8 GB 9802130 W GB9802130 W GB 9802130W WO 9903981 A8 WO9903981 A8 WO 9903981A8
Authority
WO
WIPO (PCT)
Prior art keywords
gene
nucleic acid
untranslated region
acid expression
natural position
Prior art date
Application number
PCT/GB1998/002130
Other languages
French (fr)
Other versions
WO1999003981A1 (en
Inventor
Alan Colman
Ian Garner
Michael Alexander Dalrymple
Original Assignee
Ppl Therapeutics Scotland Ltd
Alan Colman
Ian Garner
Michael Alexander Dalrymple
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 Ppl Therapeutics Scotland Ltd, Alan Colman, Ian Garner, Michael Alexander Dalrymple filed Critical Ppl Therapeutics Scotland Ltd
Priority to EP98935143A priority Critical patent/EP0996715A1/en
Priority to AU84502/98A priority patent/AU8450298A/en
Publication of WO1999003981A1 publication Critical patent/WO1999003981A1/en
Publication of WO1999003981A8 publication Critical patent/WO1999003981A8/en

Links

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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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/01—Animal expressing industrially exogenous proteins
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Environmental Sciences (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Cell Biology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

A nucleic acid expression construct comprising: (a) a promoter; (b) an intron whose natural position is within the 5'-untranslated region of a gene from which it is derived; (c) a coding sequence; and (d) a 3'-flanking sequence wherein the intron (b) is not derived from the same gene as that from which either the promoter (a) or the protein-coding sequence (c) is derived and processes, vectors, hosts and uses involving such a construct to obtain inter alia an increase in the level of expression of the coding sequence.
PCT/GB1998/002130 1997-07-17 1998-07-17 Nucleic acid expression constructs incorporating a heterologous intron whose natural position is within the 5'untranslated region of its gene WO1999003981A1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP98935143A EP0996715A1 (en) 1997-07-17 1998-07-17 Heterologous expression of proteins by "rescued" vector comprising an intron
AU84502/98A AU8450298A (en) 1997-07-17 1998-07-17 Heterologous expression of proteins by "rescued" vector comprising an intron

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB9715064.3 1997-07-17
GB9715064A GB9715064D0 (en) 1997-07-17 1997-07-17 Protein expression

Publications (2)

Publication Number Publication Date
WO1999003981A1 WO1999003981A1 (en) 1999-01-28
WO1999003981A8 true WO1999003981A8 (en) 1999-04-22

Family

ID=10816004

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB1998/002130 WO1999003981A1 (en) 1997-07-17 1998-07-17 Nucleic acid expression constructs incorporating a heterologous intron whose natural position is within the 5'untranslated region of its gene

Country Status (4)

Country Link
EP (1) EP0996715A1 (en)
AU (1) AU8450298A (en)
GB (1) GB9715064D0 (en)
WO (1) WO1999003981A1 (en)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE452973T1 (en) 1999-03-04 2010-01-15 Revivicor Inc GENETIC MODIFICATION OF SOMATIC CELLS AND THEIR USES
NZ546554A (en) * 2003-10-10 2009-04-30 Powderject Vaccines Inc Nucleic acid constructs
US9175312B2 (en) 2011-12-08 2015-11-03 Virovek Incorporation Vectors harboring toxic genes, methods and uses therefor
MX2020008133A (en) 2018-02-01 2020-10-14 Homology Medicines Inc ADENO-ASSOCIATED VIRUS COMPOSITIONS TO RESTORE PAH GENE FUNCTION AND METHODS OF USE THEREOF.
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112041437A (en) 2018-02-19 2020-12-04 同源药物公司 Adeno-Associated Virus Compositions for Restoring F8 Gene Function and Methods of Using the Same
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
CN118086341B (en) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 Expression cassette for high expression of human glucocerebrosidase gene in liver

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0174608A1 (en) * 1984-09-13 1986-03-19 The Board Of Trustees Of The Leland Stanford Junior University Beta-actin gene and regulatory elements, preparation and use
GB8615942D0 (en) * 1986-06-30 1986-08-06 Animal & Food Research Council Peptide production
EP0279582A3 (en) * 1987-02-17 1989-10-18 Pharming B.V. Dna sequences to target proteins to the mammary gland for efficient secretion
GB8717430D0 (en) * 1987-07-23 1987-08-26 Celltech Ltd Recombinant dna product
JPH02261386A (en) * 1989-03-31 1990-10-24 Kyowa Hakko Kogyo Co Ltd Recombinant vector
US6270989B1 (en) * 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5298422A (en) * 1991-11-06 1994-03-29 Baylor College Of Medicine Myogenic vector systems
AU672409B2 (en) * 1992-04-30 1996-10-03 Baylor College Of Medicine Development of a vector to target gene expression to the epidermis of transgenic animals
JP3670003B2 (en) * 1992-06-15 2005-07-13 ファーミング ビー．ブイ． Production of recombinant polypeptides by bovine species and transgenic methods
US5639940A (en) * 1994-03-03 1997-06-17 Pharmaceutical Proteins Ltd. Production of fibrinogen in transgenic animals
IL115873A0 (en) * 1995-11-03 1996-01-31 Peri Dev Applic 1985 Ltd Transgenic protein production
WO1997020043A1 (en) * 1995-11-30 1997-06-05 Zymogenetics, Inc. Protein c production in transgenic animals

Also Published As

Publication number Publication date
AU8450298A (en) 1999-02-10
WO1999003981A1 (en) 1999-01-28
EP0996715A1 (en) 2000-05-03
GB9715064D0 (en) 1997-09-24

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