JP2023535351A - 関節機能を改善する遺伝子編集 - Google Patents

関節機能を改善する遺伝子編集 Download PDF

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JP2023535351A
JP2023535351A JP2023502921A JP2023502921A JP2023535351A JP 2023535351 A JP2023535351 A JP 2023535351A JP 2023502921 A JP2023502921 A JP 2023502921A JP 2023502921 A JP2023502921 A JP 2023502921A JP 2023535351 A JP2023535351 A JP 2023535351A
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gene
guide rna
exon
sequence
nucleotides
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JP2023535351A5 (https=
Inventor
ミレット，ピーター・ジェイ
ラッセル，イアン・アラスダー
アレン，マシュー・ジェイ
ゲンチュ，ジョージ
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オーソバイオ・セラピューティクス，インコーポレイテッド
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Publication of JP2023535351A publication Critical patent/JP2023535351A/ja
Publication of JP2023535351A5 publication Critical patent/JP2023535351A5/ja
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N15/11—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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
JP2023502921A 2020-07-16 2021-07-16 関節機能を改善する遺伝子編集 Pending JP2023535351A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063052881P 2020-07-16 2020-07-16
US63/052,881 2020-07-16
US202063055808P 2020-07-23 2020-07-23
US63/055,808 2020-07-23
PCT/US2021/042100 WO2022016121A2 (en) 2020-07-16 2021-07-16 Gene editing to improve joint function

Publications (2)

Publication Number Publication Date
JP2023535351A true JP2023535351A (ja) 2023-08-17
JP2023535351A5 JP2023535351A5 (https=) 2025-01-20

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JP2023502921A Pending JP2023535351A (ja) 2020-07-16 2021-07-16 関節機能を改善する遺伝子編集

Country Status (11)

Country Link
US (1) US20230257779A1 (https=)
EP (1) EP4182461A4 (https=)
JP (1) JP2023535351A (https=)
KR (1) KR20230041729A (https=)
CN (1) CN116113696A (https=)
AU (1) AU2021308079A1 (https=)
BR (1) BR112023000738A2 (https=)
CA (1) CA3186119A1 (https=)
IL (1) IL299846A (https=)
MX (1) MX2023000671A (https=)
WO (2) WO2022016100A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4694913A2 (en) * 2023-04-11 2026-02-18 Orthobio Therapeutics, Inc. Lipid nanoparticles for gene editing systems
WO2025024814A2 (en) * 2023-07-26 2025-01-30 Orthobio Therapeutics, Inc. Transmembrane receptor gene editing
CN117448379A (zh) * 2023-12-22 2024-01-26 上海元戊医学技术有限公司 iPSC来源的IL-10蛋白过表达MSC细胞株的构建方法及其应用

Family Cites Families (7)

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Publication number Priority date Publication date Assignee Title
GB9711040D0 (en) * 1997-05-29 1997-07-23 Duff Gordon W Prediction of inflammatory disease
WO2005077333A2 (en) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
US20160279202A1 (en) * 2013-10-31 2016-09-29 Ramot At Tel-Aviv University Ltd. Interleukin-1 (il-1) inhibitors for treating fertility disorders
US20170240901A1 (en) * 2014-02-26 2017-08-24 The University Of Liverpool Nucleic acids, and uses therof
US20170335331A1 (en) * 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
WO2017223107A1 (en) * 2016-06-20 2017-12-28 Unity Biotechnology, Inc. Genome modifying enzyme therapy for diseases modulated by senescent cells
WO2018031950A1 (en) * 2016-08-12 2018-02-15 Caribou Biosciences, Inc. Protein engineering methods

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LAN ZHAO ET AL.: ""Exploration ofCRISPR/Cas9-based gene editing as therapy for osteoarthritis"", ANNALS OF THE RHEUMATIC DISEASES, vol. 78, no. 5, JPN6025037787, May 2019 (2019-05-01), pages 676 - 682, ISSN: 0005685613 *
MASATAKE ASANO: ""The Function of IL-1α a Typical Alarmin, Alarmin Interleukin-1αの機能について"", JAPANESE JOURNAL OF ORAL DIAGNOSIS / ORAL MEDICINE, vol. 33, no. 3, JPN6025037786, 2020, pages 189 - 194, ISSN: 0005685614 *

Also Published As

Publication number Publication date
IL299846A (en) 2023-03-01
WO2022016100A2 (en) 2022-01-20
US20230257779A1 (en) 2023-08-17
WO2022016100A3 (en) 2022-03-17
MX2023000671A (es) 2023-05-16
BR112023000738A2 (pt) 2023-03-21
CN116113696A (zh) 2023-05-12
AU2021308079A8 (en) 2023-03-16
WO2022016121A3 (en) 2022-03-10
EP4182461A4 (en) 2025-03-19
KR20230041729A (ko) 2023-03-24
WO2022016121A2 (en) 2022-01-20
AU2021308079A1 (en) 2023-03-02
EP4182461A2 (en) 2023-05-24
CA3186119A1 (en) 2022-01-20

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