JP2022541070A - 調節可能な発現系 - Google Patents

調節可能な発現系 Download PDF

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JP2022541070A
JP2022541070A JP2022504139A JP2022504139A JP2022541070A JP 2022541070 A JP2022541070 A JP 2022541070A JP 2022504139 A JP2022504139 A JP 2022504139A JP 2022504139 A JP2022504139 A JP 2022504139A JP 2022541070 A JP2022541070 A JP 2022541070A
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seq
nucleic acid
aav
vector
acid molecule
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JPWO2021014428A5 (https=
JP2022541070A5 (https=
Inventor
ベイベル，マーティン
ガブザー，ケラー，キャロライン
ルカシェブ，ドミトリー
ルノー，ニコル
ルディンスキー，ニキタ
シヴァサンカラン，ラジーヴ
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ノバルティス アーゲー
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Priority to JP2025013522A priority Critical patent/JP2025069281A/ja
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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
    • A61K48/0008—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
    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
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    • C12N5/0622—Glial cells, e.g. astrocytes, oligodendrocytes; Schwann cells
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/60—Vectors containing traps for, e.g. exons, promoters
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • C—CHEMISTRY; METALLURGY
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/002—Vectors comprising a special translation-regulating system controllable or inducible

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  • Animal Behavior & Ethology (AREA)
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  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022504139A 2019-07-25 2020-07-24 調節可能な発現系 Withdrawn JP2022541070A (ja)

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US201962878514P 2019-07-25 2019-07-25
US62/878,514 2019-07-25
US202063013283P 2020-04-21 2020-04-21
US63/013,283 2020-04-21
PCT/IB2020/057038 WO2021014428A1 (en) 2019-07-25 2020-07-24 Regulatable expression systems

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JP2022541070A true JP2022541070A (ja) 2022-09-21
JPWO2021014428A5 JPWO2021014428A5 (https=) 2023-08-01
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US (1) US20220307053A1 (https=)
EP (1) EP4004213A1 (https=)
JP (2) JP2022541070A (https=)
KR (1) KR20220035937A (https=)
CN (2) CN114174514A (https=)
AU (1) AU2020319168B2 (https=)
CA (1) CA3147574A1 (https=)
IL (1) IL289711A (https=)
WO (1) WO2021014428A1 (https=)

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WO2023219533A1 (ru) * 2022-05-11 2023-11-16 Общество С Ограниченной Ответственностью "Герофарм" Аденоассоциированный вирусный вектор на основе серотипа aav.php.b и его применение
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AU2020319168B2 (en) 2024-02-29
CN114174514A (zh) 2022-03-11
JP2025069281A (ja) 2025-04-30
CN120137971A (zh) 2025-06-13
WO2021014428A1 (en) 2021-01-28
IL289711A (en) 2022-03-01
KR20220035937A (ko) 2022-03-22
AU2020319168A1 (en) 2022-02-03
CA3147574A1 (en) 2021-01-28
US20220307053A1 (en) 2022-09-29

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