CA2483980C - Non-invasive delivery of polypeptides through the blood-brain barrier, and in vivo selection of endocytotic ligands - Google Patents

Non-invasive delivery of polypeptides through the blood-brain barrier, and in vivo selection of endocytotic ligands Download PDF

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Publication number
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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Prior art keywords
neurons
vector
polypeptide
bbb
blood
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Expired - Fee Related
Application number
CA2483980A
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English (en)
French (fr)
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CA2483980A1 (en
Inventor
Ian A. Ferguson
Hiroaki Tani
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Individual
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Publication date
Priority claimed from AUPS1935A external-priority patent/AUPS193502A0/en
Priority claimed from US10/188,184 external-priority patent/US20030083299A1/en
Application filed by Individual filed Critical Individual
Publication of CA2483980A1 publication Critical patent/CA2483980A1/en
Application granted granted Critical
Publication of CA2483980C publication Critical patent/CA2483980C/en
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/005Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies constructed by phage libraries
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2878Immunoglobulins [IGs], 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/21Immunoglobulins specific features characterized by taxonomic origin from primates, e.g. man
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16611Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use 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)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (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)
CA2483980A 2002-04-26 2003-04-28 Non-invasive delivery of polypeptides through the blood-brain barrier, and in vivo selection of endocytotic ligands Expired - Fee Related CA2483980C (en)

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 2002-07-02
US10/188,184 US20030083299A1 (en) 2000-11-04 2002-07-02 Non-invasive delivery of polypeptides through the blood-brain barrier
PCT/IB2003/002371 WO2003091387A2 (en) 2002-04-26 2003-04-28 Non-invasive delivery of polypeptides through the blood-brain barrier, and in vivo selection of endocytotic ligands

Publications (2)

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

Family

ID=29271266

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2483980A Expired - Fee Related CA2483980C (en) 2002-04-26 2003-04-28 Non-invasive delivery of polypeptides through the blood-brain barrier, and in vivo selection of endocytotic ligands

Country Status (4)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006070290A2 (en) * 2004-06-23 2006-07-06 Ferguson Ian A Agents and methods for early diagnosis and monitoring of alzheimer's disease and other neurological disorders
EP1985311A4 (en) * 2006-01-24 2011-04-13 Univ Kagoshima AGENTS FOR TARGETING A MEDICAMENT ON CEREBRAL NEURONS
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 (en) 2006-02-08
WO2003091387A2 (en) 2003-11-06
WO2003091387A3 (en) 2004-01-08
WO2003091387A9 (en) 2004-02-26
JP2005535580A (ja) 2005-11-24
JP2013136586A (ja) 2013-07-11
CA2483980A1 (en) 2003-11-06
EP1503801A2 (en) 2005-02-09

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