WO2019103442A3 - CRISPR/Cpf1 시스템을 이용한 유전체 편집용 조성물 및 이의 용도 - Google Patents

CRISPR/Cpf1 시스템을 이용한 유전체 편집용 조성물 및 이의 용도 Download PDF

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WO2019103442A3
WO2019103442A3 PCT/KR2018/014312 KR2018014312W WO2019103442A3 WO 2019103442 A3 WO2019103442 A3 WO 2019103442A3 KR 2018014312 W KR2018014312 W KR 2018014312W WO 2019103442 A3 WO2019103442 A3 WO 2019103442A3
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genome editing
crispr
cpf1 system
editing composition
cpf1
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French (fr)
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WO2019103442A2 (ko
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김용삼
고정헌
이정미
문수빈
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한국생명공학연구원
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Priority to CN201880085616.XA priority Critical patent/CN111836894B/zh
Priority to AU2018370588A priority patent/AU2018370588B2/en
Priority to EP18882084.9A priority patent/EP3715461A4/en
Priority to US16/765,553 priority patent/US11667917B2/en
Publication of WO2019103442A2 publication Critical patent/WO2019103442A2/ko
Publication of WO2019103442A3 publication Critical patent/WO2019103442A3/ko

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Abstract

본 발명은 CRISPR/Cpf1 시스템을 이용한 유전체 편집용 조성물 및 이의 용도에 관한 것으로서, 보다 상세하게 본 발명은 표적 뉴클레오티드(target nucleotide) 서열과 혼성화 가능한 가이드 서열(guide sequence) 및 상기 가이드 서열의 3'-말단에 연결된 유리딘(U, uridine) 반복 서열을 포함하는 crRNA(CRISPR RNA) 또는 이를 암호화하는 DNA; 및 Cpf1 단백질 또는 이를 암호화하는 DNA을 포함하는 유전체 편집용 조성물, 이를 이용한 유전체 편집 방법, 형질 전환체 제조 방법 및 형질 전환체에 관한 것이다. 본 발명은 CRISPR/Cpf1 시스템을 이용하여 진핵 세포의 유전제 교정을 수행함에 있어서 인델 효율을 높이고 오프-타겟 활성을 줄일 수 있고, 원하는 유전자가 삽입(knock-in) 또는 결실(knock-out)된 형질 전환 세포, 형질 전환 동물 또는 형질 전환 식물을 용이하게 제조할 수 있다.
PCT/KR2018/014312 2017-11-21 2018-11-21 CRISPR/Cpf1 시스템을 이용한 유전체 편집용 조성물 및 이의 용도 WO2019103442A2 (ko)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CN201880085616.XA CN111836894B (zh) 2017-11-21 2018-11-21 用于使用CRISPR/Cpf1***进行基因组编辑的组合物及其用途
AU2018370588A AU2018370588B2 (en) 2017-11-21 2018-11-21 Composition for Genome Editing Using Crispr/CPF1 System and Use Thereof
EP18882084.9A EP3715461A4 (en) 2017-11-21 2018-11-21 GENOMIC EDITION COMPOSITION USING A CRISPR / CPF1 SYSTEM AND ITS USE
US16/765,553 US11667917B2 (en) 2017-11-21 2018-11-21 Composition for genome editing using CRISPR/CPF1 system and use thereof

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KR20170155927 2017-11-21
KR10-2017-0155927 2017-11-21

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US (1) US11667917B2 (ko)
EP (1) EP3715461A4 (ko)
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WO (1) WO2019103442A2 (ko)

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US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN114846146B (zh) * 2019-10-29 2024-04-12 基恩科雷有限责任公司 用于增加CRISPR/Cas12f1***的效率的工程化引导RNA及其用途
WO2021092519A1 (en) * 2019-11-08 2021-05-14 University Of Florida Research Foundation Crispr complex-based detection system and method
CN111235130B (zh) * 2019-11-15 2022-11-25 武汉大学 II类V型CRISPR蛋白CeCas12a及其在基因编辑的应用
KR102551876B1 (ko) * 2019-12-18 2023-07-05 한국생명공학연구원 Cpf1 및 키메릭 DNA-RNA 가이드를 포함하는 유전체 교정 또는 발현 억제용 조성물
KR102493512B1 (ko) * 2020-10-08 2023-02-01 주식회사 진코어 CRISPR/Cas12a 시스템을 위한 엔지니어링 된 crRNA
CN116568803A (zh) * 2020-10-08 2023-08-08 基恩科雷有限责任公司 用于增加CRISPR/Cas12f1***的效率的包含富含U的尾部的经工程化的引导RNA及其用途
CA3220294A1 (en) * 2021-05-14 2022-11-17 Genkore Inc. Composition and method for treatment of lca10 using rna-guided nuclease
CN113355362B (zh) * 2021-06-16 2022-06-07 北京大学 化学修饰CRISPR/Cpf1复合物的用途
KR20230121569A (ko) * 2022-02-09 2023-08-18 주식회사 진코어 상동지정복구를 위한 TaRGET 시스템 및 이를 이용한 유전자 편집 방법
WO2023172116A1 (ko) * 2022-03-10 2023-09-14 주식회사 진코어 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법
KR20230142365A (ko) * 2022-03-30 2023-10-11 주식회사 진코어 어셔 증후군 치료를 위한 유전자 편집 시스템
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KR102168489B1 (ko) 2020-10-22
EP3715461A4 (en) 2021-09-08
CN111836894A (zh) 2020-10-27
AU2018370588A1 (en) 2020-07-09
EP3715461A2 (en) 2020-09-30
US20200308583A1 (en) 2020-10-01
KR20190058358A (ko) 2019-05-29
AU2018370588B2 (en) 2022-09-08
US11667917B2 (en) 2023-06-06
CN111836894B (zh) 2023-11-10

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