JP2023515795A - Ａａｖカプシド－プロモーター相互作用および細胞選択的遺伝子発現 - Google Patents

Ａａｖカプシド－プロモーター相互作用および細胞選択的遺伝子発現 Download PDF

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JP2023515795A
JP2023515795A JP2022549613A JP2022549613A JP2023515795A JP 2023515795 A JP2023515795 A JP 2023515795A JP 2022549613 A JP2022549613 A JP 2022549613A JP 2022549613 A JP2022549613 A JP 2022549613A JP 2023515795 A JP2023515795 A JP 2023515795A
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promoter
aav
amino acid
capsid protein
derivative
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Japanese (ja)
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JPWO2021167919A5 (https=
JP2023515795A5 (https=
Inventor
マカウン，トーマス
パウエル，サラ
ジュード サミュルスキー，リチャード
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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Publication of JP2023515795A5 publication Critical patent/JP2023515795A5/ja
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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered 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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
JP2022549613A 2020-02-18 2021-02-17 Ａａｖカプシド－プロモーター相互作用および細胞選択的遺伝子発現 Pending JP2023515795A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062977838P 2020-02-18 2020-02-18
US62/977,838 2020-02-18
US202063023003P 2020-05-11 2020-05-11
US63/023,003 2020-05-11
PCT/US2021/018280 WO2021167919A1 (en) 2020-02-18 2021-02-17 Aav capsid-promoter interactions and cell selective gene expression

Publications (3)

Publication Number Publication Date
JP2023515795A true JP2023515795A (ja) 2023-04-14
JPWO2021167919A5 JPWO2021167919A5 (https=) 2024-02-27
JP2023515795A5 JP2023515795A5 (https=) 2024-02-27

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JP2022549613A Pending JP2023515795A (ja) 2020-02-18 2021-02-17 Ａａｖカプシド－プロモーター相互作用および細胞選択的遺伝子発現

Country Status (7)

Country Link
US (1) US20230013145A1 (https=)
EP (1) EP4107169A4 (https=)
JP (1) JP2023515795A (https=)
CN (1) CN115210248A (https=)
AU (1) AU2021224551A1 (https=)
CA (1) CA3164321A1 (https=)
WO (1) WO2021167919A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250206783A1 (en) * 2022-03-21 2025-06-26 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism
WO2023201306A1 (en) 2022-04-14 2023-10-19 Invisishield Technologies Ltd. Compositions for preventing or treating influenza infections
WO2025230969A1 (en) * 2024-05-02 2025-11-06 4D Molecular Therapeutics Inc. Aav variants with improved cns tropism and uses thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004099423A1 (en) * 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vectors and uses thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003221733A1 (en) * 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
WO2010071454A1 (en) * 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
WO2016081811A1 (en) * 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004099423A1 (en) * 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vectors and uses thereof

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
DATABASE GENBANK [ONLINE], JPN6025004563, pages 70938, ISSN: 0005656627 *
DATABASE GENBANK [ONLINE], JPN6025004564, pages 69527, ISSN: 0005656628 *
DATABASE GENBANK [ONLINE], JPN6025004565, pages 45836, ISSN: 0005656629 *
PEI WU ET AL.: ""Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AA", JOURNAL OF VIROLOGY, vol. 74, no. 18, JPN6025004566, 15 September 2000 (2000-09-15), pages 8635 - 8647, ISSN: 0005656630 *

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Publication number Publication date
US20230013145A1 (en) 2023-01-19
EP4107169A1 (en) 2022-12-28
AU2021224551A1 (en) 2022-07-28
CN115210248A (zh) 2022-10-18
WO2021167919A1 (en) 2021-08-26
CA3164321A1 (en) 2021-08-26
EP4107169A4 (en) 2024-07-17

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