JP7628319B2 - ｉｎ ｖｉｖｏ遺伝子編集のための組成物および方法 - Google Patents

ｉｎ ｖｉｖｏ遺伝子編集のための組成物および方法 Download PDF

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JP7628319B2
JP7628319B2 JP2022511242A JP2022511242A JP7628319B2 JP 7628319 B2 JP7628319 B2 JP 7628319B2 JP 2022511242 A JP2022511242 A JP 2022511242A JP 2022511242 A JP2022511242 A JP 2022511242A JP 7628319 B2 JP7628319 B2 JP 7628319B2
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cells
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composition
gene
cell
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ベルモンテ， ジュアン カルロス イズピスア
啓一郎 鈴木
真子 辻
レイナ ヘルナンデス－ベニテス，
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ソーク インスティテュート フォー バイオロジカル スタディーズ
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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6844—Nucleic acid amplification reactions
    • C12Q1/686—Polymerase chain reaction [PCR]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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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    • 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
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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
    • C12N2800/40—Systems of functionally co-operating vectors

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  • Engineering & Computer Science (AREA)
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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
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  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022511242A 2019-08-22 2019-09-20 ｉｎ ｖｉｖｏ遺伝子編集のための組成物および方法 Active JP7628319B2 (ja)

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JP2024193935A JP2025016714A (ja) 2019-08-22 2024-11-05 ｉｎ ｖｉｖｏ遺伝子編集のための組成物および方法

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US201962890542P 2019-08-22 2019-08-22
US62/890,542 2019-08-22
US201962891210P 2019-08-23 2019-08-23
US62/891,210 2019-08-23
PCT/US2019/052266 WO2021034336A1 (en) 2019-08-22 2019-09-20 Compositions and methods for in vivo gene editing

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JP7628319B2 true JP7628319B2 (ja) 2025-02-10

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US (1) US20220333106A1 (enExample)
EP (1) EP4017543A4 (enExample)
JP (2) JP7628319B2 (enExample)
AU (1) AU2019462506A1 (enExample)
CA (1) CA3152056A1 (enExample)
WO (1) WO2021034336A1 (enExample)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220333106A1 (en) * 2019-08-22 2022-10-20 Salk Institute For Biological Studies Compositions and methods for in vivo gene editing
CN116200368A (zh) * 2021-11-30 2023-06-02 上海科技大学 一种基于c2c9核酸酶的新型基因组编辑系统及其应用
WO2023187779A1 (en) * 2022-03-28 2023-10-05 Ramot At Tel-Aviv University Ltd. Site-specific in vivo t cell engineering, systems, compositions and methods thereof
CN118006739B (zh) * 2024-02-05 2024-10-29 北京亿森宝生物科技有限公司 一种同时检测禽滑液囊支原体野毒和疫苗的方法、产品及应用
CN119685398B (zh) * 2025-02-26 2025-05-23 北京林业大学 一种敲除牛fbxo40基因的方法及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018013932A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells

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US20120196370A1 (en) * 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
US11939571B2 (en) * 2016-06-10 2024-03-26 President And Fellows Of Harvard College Library-scale engineering of metabolic pathways
US20220333106A1 (en) * 2019-08-22 2022-10-20 Salk Institute For Biological Studies Compositions and methods for in vivo gene editing

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018013932A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Basiri, M. et al.，"The Convenience of Single Homology Arm Donor DNA and CRISPR/Cas9-Nickase for Targeted Insertion of Long DNA Fragment"，Cell J.，2017年，Vol. 18，pp. 532-539
Suzuki, K. et al.，"Development of in vivo genome editing technology, HITI, and application for genome-editing therapy"，日本ゲノム編集学会第４回大会 要旨集，2019年06月03日，p. 48

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AU2019462506A1 (en) 2022-03-24
EP4017543A4 (en) 2025-01-22
US20220333106A1 (en) 2022-10-20
CA3152056A1 (en) 2021-02-25
EP4017543A1 (en) 2022-06-29
JP2022553607A (ja) 2022-12-26
WO2021034336A1 (en) 2021-02-25
JP2025016714A (ja) 2025-02-04

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