KR20240116827A - 신규한 crispr 효소, 방법, 시스템 및 이의 용도 - Google Patents

신규한 crispr 효소, 방법, 시스템 및 이의 용도 Download PDF

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Publication number
KR20240116827A
KR20240116827A KR1020247023120A KR20247023120A KR20240116827A KR 20240116827 A KR20240116827 A KR 20240116827A KR 1020247023120 A KR1020247023120 A KR 1020247023120A KR 20247023120 A KR20247023120 A KR 20247023120A KR 20240116827 A KR20240116827 A KR 20240116827A
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South Korea
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sequence
cas9
strain
cas9 protein
nucleic acid
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KR1020247023120A
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English (en)
Korean (ko)
Inventor
베른트 제체
데이비드 에이. 본
루이스 바레라
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빔 테라퓨틱스, 인크.
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
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    • 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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    • C12N15/63—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
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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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • 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)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
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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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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • General Health & Medical Sciences (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020247023120A 2021-12-17 2022-12-16 신규한 crispr 효소, 방법, 시스템 및 이의 용도 Pending KR20240116827A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163291252P 2021-12-17 2021-12-17
US63/291,252 2021-12-17
PCT/US2022/081728 WO2023114953A2 (en) 2021-12-17 2022-12-16 Novel crispr enzymes, methods, systems and uses thereof

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KR20240116827A true KR20240116827A (ko) 2024-07-30

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KR1020247023120A Pending KR20240116827A (ko) 2021-12-17 2022-12-16 신규한 crispr 효소, 방법, 시스템 및 이의 용도

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US (1) US20240327813A1 (https=)
EP (1) EP4448772A2 (https=)
JP (1) JP2024546841A (https=)
KR (1) KR20240116827A (https=)
CN (1) CN119095968A (https=)
AU (1) AU2022413670A1 (https=)
CA (1) CA3240917A1 (https=)
WO (1) WO2023114953A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121079412A (zh) 2023-04-28 2025-12-05 比姆医疗股份有限公司 经修饰的指导rna
CN121294400A (zh) * 2024-07-08 2026-01-09 华熙生物科技股份有限公司 一种Cas蛋白及其基因编辑系统和应用
CN119876089A (zh) * 2025-01-10 2025-04-25 上海市浦东医院（复旦大学附属浦东医院） 基于SeqCas9蛋白的CRISPR/Cas9基因编辑系统及其相关应用

Family Cites Families (16)

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US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
SG11201906297QA (en) * 2017-03-24 2019-10-30 Curevac Ag Nucleic acids encoding crispr-associated proteins and uses thereof

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CN119095968A (zh) 2024-12-06
WO2023114953A3 (en) 2023-07-27
CA3240917A1 (en) 2023-06-22
US20240327813A1 (en) 2024-10-03
WO2023114953A2 (en) 2023-06-22
AU2022413670A1 (en) 2024-07-04
JP2024546841A (ja) 2024-12-26
EP4448772A2 (en) 2024-10-23

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