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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JP2023515795A5 (enExample
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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    • 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
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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
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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)
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  • Engineering & Computer Science (AREA)
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  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (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 (2)

Publication Number Publication Date
JP2023515795A true JP2023515795A (ja) 2023-04-14
JP2023515795A5 JP2023515795A5 (enExample) 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

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US (1) US20230013145A1 (enExample)
EP (1) EP4107169A4 (enExample)
JP (1) JP2023515795A (enExample)
CN (1) CN115210248A (enExample)
AU (1) AU2021224551A1 (enExample)
CA (1) CA3164321A1 (enExample)
WO (1) WO2021167919A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023183304A2 (en) * 2022-03-21 2023-09-28 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism
EP4508077A4 (en) 2022-04-14 2026-03-25 Invisishield Tech Ltd COMPOSITIONS FOR PREVENTING OR TREATMENT OF 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
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
AU2015349759B2 (en) * 2014-11-21 2022-01-06 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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EP4107169A4 (en) 2024-07-17
CN115210248A (zh) 2022-10-18
US20230013145A1 (en) 2023-01-19
CA3164321A1 (en) 2021-08-26
EP4107169A1 (en) 2022-12-28
WO2021167919A1 (en) 2021-08-26
AU2021224551A1 (en) 2022-07-28

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