JP7606176B2 - ゲノム編集効率を向上させる融合タンパク質及びその使用 - Google Patents

ゲノム編集効率を向上させる融合タンパク質及びその使用 Download PDF

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JP7606176B2
JP7606176B2 JP2022538379A JP2022538379A JP7606176B2 JP 7606176 B2 JP7606176 B2 JP 7606176B2 JP 2022538379 A JP2022538379 A JP 2022538379A JP 2022538379 A JP2022538379 A JP 2022538379A JP 7606176 B2 JP7606176 B2 JP 7606176B2
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fusion protein
cell
seq
be4max
sequence
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JP2023507034A5 (https=
JP2023507034A (ja
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リー、ターリー
チャン、シアオホイ
リウ、ミンヤオ
チュー、ピーユン
チェン、リャン
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BRL Medicine Inc
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BRL Medicine Inc
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Priority claimed from CN201911310969.8A external-priority patent/CN112979821B/zh
Priority claimed from CN201911312544.0A external-priority patent/CN112979823B/zh
Priority claimed from CN201911312537.0A external-priority patent/CN112979822B/zh
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C12N15/87—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
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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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]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04001—Cytosine deaminase (3.5.4.1)
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    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
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    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • 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
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    • 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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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C07K—PEPTIDES
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    • C12N2310/00—Structure or type of the nucleic acid
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JP2022538379A 2019-12-18 2020-12-17 ゲノム編集効率を向上させる融合タンパク質及びその使用 Active JP7606176B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
CN201911310969.8 2019-12-18
CN201911310969.8A CN112979821B (zh) 2019-12-18 2019-12-18 一种提高基因编辑效率的融合蛋白及其应用
CN201911312544.0A CN112979823B (zh) 2019-12-18 2019-12-18 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白
CN201911312537.0 2019-12-18
CN201911312544.0 2019-12-18
CN201911312537.0A CN112979822B (zh) 2019-12-18 2019-12-18 一种疾病动物模型的构建方法及融合蛋白
PCT/CN2020/137239 WO2021121321A1 (zh) 2019-12-18 2020-12-17 一种提高基因编辑效率的融合蛋白及其应用

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JP2023507034A JP2023507034A (ja) 2023-02-20
JP2023507034A5 JP2023507034A5 (https=) 2024-03-26
JP7606176B2 true JP7606176B2 (ja) 2024-12-25

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KR102616916B1 (ko) * 2021-05-11 2023-12-21 주식회사 애이마 발암성 돌연변이 유전자 검출용 가이드 rna 내지 이의 용도
CN113621634B (zh) * 2021-07-07 2023-09-15 浙江大学杭州国际科创中心 一种增加基因组突变率的碱基编辑系统及碱基编辑方法
JP7125727B1 (ja) * 2021-09-07 2022-08-25 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法

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WO2010132092A2 (en) 2009-05-12 2010-11-18 The Scripps Research Institute Cytidine deaminase fusions and related methods
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods

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ITFI20120173A1 (it) * 2012-08-30 2014-03-01 Selex Galileo Spa "sistema di visione con telecamera e sistema di limitazione delle sollecitazioni"
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP6799586B2 (ja) * 2015-08-28 2020-12-16 ザ ジェネラル ホスピタル コーポレイション 遺伝子操作ＣＲＩＳＰＲ−Ｃａｓ９ヌクレアーゼ
CN105647968B (zh) * 2016-02-02 2019-07-23 浙江大学 一种CRISPR/Cas9工作效率快速测试系统及其应用
WO2017142923A1 (en) * 2016-02-16 2017-08-24 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
CN108588182B (zh) * 2018-04-13 2025-11-28 武汉中科先进技术研究院有限公司 基于crispr-链取代的等温扩增及检测技术
CN108707635B (zh) * 2018-05-29 2022-02-15 华东师范大学 用于核苷酸序列修饰的组合物、方法与应用
CN109880851B (zh) * 2019-03-28 2020-12-01 西北农林科技大学 用于富集CRISPR/Cas9介导的同源重组修复细胞的筛选报告载体及筛选方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010132092A2 (en) 2009-05-12 2010-11-18 The Scripps Research Institute Cytidine deaminase fusions and related methods
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods

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WO2021121321A1 (zh) 2021-06-24
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US20220364072A1 (en) 2022-11-17
EP4079765A4 (en) 2024-02-14

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