CA2483980C - Administration non invasive de polypeptides a travers la barriere hemato-encephalique et selection in vivo de ligands endocytotiques - Google Patents

Administration non invasive de polypeptides a travers la barriere hemato-encephalique et selection in vivo de ligands endocytotiques Download PDF

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CA2483980C
CA2483980C CA2483980A CA2483980A CA2483980C CA 2483980 C CA2483980 C CA 2483980C CA 2483980 A CA2483980 A CA 2483980A CA 2483980 A CA2483980 A CA 2483980A CA 2483980 C CA2483980 C CA 2483980C
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neurons
vector
polypeptide
bbb
blood
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CA2483980A1 (fr
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Ian A. Ferguson
Hiroaki Tani
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Priority claimed from AUPS1935A external-priority patent/AUPS193502A0/en
Priority claimed from US10/188,184 external-priority patent/US20030083299A1/en
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    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/005—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies constructed by phage libraries
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2878—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/21—Immunoglobulins specific features characterized by taxonomic origin from primates, e.g. man
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
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  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
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  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Pulmonology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention se rapporte à une méthode de traitement et à des vecteurs génétiques permettant l'administration non invasive de polypeptides à travers la barrière hémato-encéphalique (BBB), aux fins du traitement d'un tissu cérébral ou spinal. Un vecteur génétique est utilisé pour transfecter un ou plusieurs neurones qui "chevauche(nt)" la barrière BBB, tels que des neurones sensitifs, des neurones nociceptifs ou des neurones moteurs inférieurs; pour ce faire, le vecteur est administré de manière à provoquer son contact avec des protubérances neuronales qui s'étendent à l'extérieur de la BBB. Une fois à l'intérieur d'une projection périphérique qui appartient à un neurone chevauchant la BBB, les vecteurs (ou une partie de ceux-ci) sont transportés vers le corps cellulaire principal du neurone, selon un processus appelé transport rétrograde. A l'intérieur du corps cellulaire principal, au moins un gène porté par le vecteur génétique sera exprimé, de manière à former des polypeptides. Certains de ces polypeptides (qui peuvent inclure des séquences de tête qui peuvent favoriser le transport antérograde et la sécrétion par des neurones chevauchant la BBB) sont transportés par les neurones vers des sites de sécrétion à l'intérieur de la BBB. Les polypeptides sont ensuite sécrétés par les neurones transfectés en des emplacements intérieurs à la BBB, et ils entrent ensuite en contact avec des neurones secondaires "cible" et exercent leurs effets sur ces neurones situés entièrement à l'intérieur de la BBB. Grâce à ce système, les polypeptides qui stimulent la croissance ou l'activité nerveuse peuvent être utilisés pour traiter des maladies neurodégénératives, des membres endommagés chez des victimes d'accidents vasculaires cérébraux, etc., et les polypeptides qui suppriment l'activité neuronale peuvent être utilisés pour traiter une activité neuronale excessive non souhaitée, telle que la douleur neuropatique. L'invention se rapporte également à de nouvelles méthodes d'administration de polypeptides endocriniens et paracriniens au système nerveux central, ceci permettant l'amélioration de traitements médicaux et de traitements visant à favoriser la reproduction chez des sujets humains, ainsi que l'amélioration de la capacité à moduler la croissance, la maturation, la reproduction ou d'autres fonctions liées au système endocrinien sur du bétail, des espèces menacées et d'autres animaux.
CA2483980A 2002-04-26 2003-04-28 Administration non invasive de polypeptides a travers la barriere hemato-encephalique et selection in vivo de ligands endocytotiques Expired - Fee Related CA2483980C (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
AUPS1935A AUPS193502A0 (en) 2002-04-26 2002-04-26 Molecules that mediate internalisation in cells
AUPS1935 2002-04-26
US10/188,184 US20030083299A1 (en) 2000-11-04 2002-07-02 Non-invasive delivery of polypeptides through the blood-brain barrier
US10/188,184 2002-07-02
PCT/IB2003/002371 WO2003091387A2 (fr) 2002-04-26 2003-04-28 Administration non invasive de polypeptides a travers la barriere hemato-encephalique et selection in vivo de ligands endocytotiques

Publications (2)

Publication Number Publication Date
CA2483980A1 CA2483980A1 (fr) 2003-11-06
CA2483980C true CA2483980C (fr) 2013-07-16

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CA2483980A Expired - Fee Related CA2483980C (fr) 2002-04-26 2003-04-28 Administration non invasive de polypeptides a travers la barriere hemato-encephalique et selection in vivo de ligands endocytotiques

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Country Link
EP (1) EP1503801A4 (fr)
JP (2) JP2005535580A (fr)
CA (1) CA2483980C (fr)
WO (1) WO2003091387A2 (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2460969B (en) * 2004-06-23 2010-02-17 Ian A Ferguson Vaccines for intranasal administration
EP1985311A4 (fr) * 2006-01-24 2011-04-13 Univ Kagoshima Agent pour cibler un médicament sur un neurone cérébral
JP2011024499A (ja) * 2009-07-27 2011-02-10 Nec Soft Ltd 核酸の製造方法、デリバリー対象物質を臓器特異的にデリバリー可能なデリバリー用タグ核酸およびその用途
US20130123647A1 (en) 2011-05-03 2013-05-16 The Research Foundation Of State University Of New York Methods Useful in Optimizing the Treatment of Neuropathies and Targeting Tissues with Cosmetic Botulinum Injections
CN113838541B (zh) * 2021-09-29 2023-10-10 脸萌有限公司 设计配体分子的方法和装置

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6040172A (en) * 1992-08-14 2000-03-21 The Rockefeller University Defective DNA viral vector comprising a neural tissue-specific promoter for in vivo expression of a gene
US6770797B2 (en) * 2001-06-01 2004-08-03 Rhode Island Hospital Non-Transgenic nonhuman model for Alzheimer's Disease using a AD7c-NTP nucleic acid

Also Published As

Publication number Publication date
EP1503801A4 (fr) 2006-02-08
JP2013136586A (ja) 2013-07-11
WO2003091387A3 (fr) 2004-01-08
CA2483980A1 (fr) 2003-11-06
WO2003091387A2 (fr) 2003-11-06
EP1503801A2 (fr) 2005-02-09
JP2005535580A (ja) 2005-11-24
WO2003091387A9 (fr) 2004-02-26

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