WO2009122401A3 - Procédés d'expression d'un polypeptide dans le cerveau et produits de construction d'acide nucléique capables d'effectuer ces procédés - Google Patents

Procédés d'expression d'un polypeptide dans le cerveau et produits de construction d'acide nucléique capables d'effectuer ces procédés Download PDF

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Publication number
WO2009122401A3
WO2009122401A3 PCT/IL2009/000341 IL2009000341W WO2009122401A3 WO 2009122401 A3 WO2009122401 A3 WO 2009122401A3 IL 2009000341 W IL2009000341 W IL 2009000341W WO 2009122401 A3 WO2009122401 A3 WO 2009122401A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
polypeptide
same
brain
expressing
Prior art date
Application number
PCT/IL2009/000341
Other languages
English (en)
Other versions
WO2009122401A2 (fr
Inventor
Alon Chen
Original Assignee
Yeda Research And Development Co. Ltd.
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 Yeda Research And Development Co. Ltd. filed Critical Yeda Research And Development Co. Ltd.
Priority to EP09728297A priority Critical patent/EP2271763A2/fr
Priority to US12/934,689 priority patent/US20110288159A1/en
Publication of WO2009122401A2 publication Critical patent/WO2009122401A2/fr
Publication of WO2009122401A3 publication Critical patent/WO2009122401A3/fr
Priority to IL208395A priority patent/IL208395A/en

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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
    • 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
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/05—Animals modified by non-integrating nucleic acids, e.g. antisense, RNAi, morpholino, episomal vector, for non-therapeutic purpose
    • 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
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0356—Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

L'invention porte sur un produit de construction d'acide nucléique et sur un système de produit de construction. Le produit de construction d'acide nucléique et le système comprennent une séquence régulatrice qui régule l'expression inductible d'un polypeptide d'intérêt, la séquence régulatrice comprenant un promoteur spécifique du plexus choroïde, à la condition que le promoteur spécifique du plexus choroïde ne soit pas un promoteur de transthyrétine. L'invention porte également sur des compositions pharmaceutiques les comprenant et sur leurs utilisations.
PCT/IL2009/000341 2008-04-03 2009-03-25 Procédés d'expression d'un polypeptide dans le cerveau et produits de construction d'acide nucléique capables d'effectuer ces procédés WO2009122401A2 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP09728297A EP2271763A2 (fr) 2008-04-03 2009-03-25 Procédés d'expression d'un polypeptide dans le cerveau et produits de construction d'acide nucléique capables d'effectuer ces procédés
US12/934,689 US20110288159A1 (en) 2008-04-03 2009-03-25 Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same
IL208395A IL208395A (en) 2008-04-03 2010-10-03 Constructs containing choroid plexus specific promoters for the expression of neuripeptides secreted in this brain and medicinal preparations containing them

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US6493908P 2008-04-03 2008-04-03
US61/064,939 2008-04-03

Publications (2)

Publication Number Publication Date
WO2009122401A2 WO2009122401A2 (fr) 2009-10-08
WO2009122401A3 true WO2009122401A3 (fr) 2010-01-28

Family

ID=40902065

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IL2009/000341 WO2009122401A2 (fr) 2008-04-03 2009-03-25 Procédés d'expression d'un polypeptide dans le cerveau et produits de construction d'acide nucléique capables d'effectuer ces procédés

Country Status (2)

Country Link
EP (1) EP2271763A2 (fr)
WO (1) WO2009122401A2 (fr)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102206285B (zh) * 2011-04-19 2013-02-13 兰州大学 基于内吗啡肽2和神经肽ff的嵌合肽及其合成和应用
FR3007988B1 (fr) 2013-07-04 2015-09-25 Centre Nat Rech Scient Inhibition de la synthese de la proteine betaapp ou de l'activite du peptide abeta dans les plexus choroides.
WO2017180857A1 (fr) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Thérapie génique pour le traitement de l'hémophilie a

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004113493A2 (fr) * 2003-06-03 2004-12-29 Cell Genesys, Inc. Compositions et procedes permettant l'expression accrue d'immunoglobulines a partir d'un seul vecteur utilisant un site de clivage peptidique
US20060189561A1 (en) * 2005-02-16 2006-08-24 Roelvink Petrus W RNAi expression constructs with liver-specific enhancer/promoter
US20070243168A1 (en) * 2006-03-30 2007-10-18 Mark Kay Minigene expression cassette
WO2009013290A1 (fr) * 2007-07-23 2009-01-29 Genethon Administration de gène au système nerveux central par administration périphérique de vecteurs aav

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004113493A2 (fr) * 2003-06-03 2004-12-29 Cell Genesys, Inc. Compositions et procedes permettant l'expression accrue d'immunoglobulines a partir d'un seul vecteur utilisant un site de clivage peptidique
US20060189561A1 (en) * 2005-02-16 2006-08-24 Roelvink Petrus W RNAi expression constructs with liver-specific enhancer/promoter
US20070243168A1 (en) * 2006-03-30 2007-10-18 Mark Kay Minigene expression cassette
WO2009013290A1 (fr) * 2007-07-23 2009-01-29 Genethon Administration de gène au système nerveux central par administration périphérique de vecteurs aav

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
CHEN A. ET AL.: "Mouse corticotropin-releasing factor receptor type 2alpha gene: Isolation, distribution, pharmacological characterization and regulation by stress and glucocorticoids", MOLECULAR ENDOCRINOLOGY, vol. 19, no. 2, February 2005 (2005-02-01), pages 441 - 458, XP002539926, ISSN: 0888-8809 *
CHTARTO A. ET AL.: "Tetracycline-inducible transgene expression mediated by a single AAV vector.", GENE THERAPY, vol. 10, no. 1, January 2003 (2003-01-01), pages 84 - 94, XP002540413, ISSN: 0969-7128 *
NOGAMI H. ET AL.: "Region-specific expression and hormonal regulation of the first exon variants of rat prolactin receptor mRNA in rat brain and anterior pituitary gland", JOURNAL OF NEUROENDOCRINOLOGY, vol. 19, no. 8, August 2007 (2007-08-01), pages 583 - 593, XP002540106, ISSN: 0953-8194 *
OLDSTONE M.B.A. ET AL.: "Trafficking of activated cytotoxic T lymphocytes into the central nervous system: Use of a transgenic model", JOURNAL OF NEUROIMMUNOLOGY, ELSEVIER SCIENCE PUBLISHERS BV, XX, vol. 46, no. 1-2, 1 July 1993 (1993-07-01), pages 25 - 31, XP023694949, ISSN: 0165-5728, [retrieved on 19930701] *
PICKERING C. ET AL.: "The Adhesion GPCR GPR125 is specifically expressed in the choroid plexus and is upregulated following brain injury", BMC NEUROSCIENCE, BIOMED CENTRAL, LONDON, GB, vol. 9, no. 1, 3 October 2008 (2008-10-03), pages 97, XP021042488, ISSN: 1471-2202 *
SAENZ-ROBLES M.T. ET AL.: "Induction versus progression of brain tumor development: Differential functions for the pRB- and p53-targeting domains of simian virus 40 T antigen", MOLECULAR AND CELLULAR BIOLOGY, vol. 14, no. 4, 1994, pages 2686 - 2698, XP002540107, ISSN: 0270-7306 *

Also Published As

Publication number Publication date
WO2009122401A2 (fr) 2009-10-08
EP2271763A2 (fr) 2011-01-12

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