WO2019117660A3 - Crispr 시스템 기능 향상 방법 및 그의 이용 - Google Patents

Crispr 시스템 기능 향상 방법 및 그의 이용 Download PDF

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WO2019117660A3
WO2019117660A3 PCT/KR2018/015897 KR2018015897W WO2019117660A3 WO 2019117660 A3 WO2019117660 A3 WO 2019117660A3 KR 2018015897 W KR2018015897 W KR 2018015897W WO 2019117660 A3 WO2019117660 A3 WO 2019117660A3
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sgrna
crispr system
same
compositions
development
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WO2019117660A2 (ko
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이성욱
이창호
한승렬
김지현
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단국대학교 산학협력단
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Abstract

본 발명은 특이적 sgRNA를 이용하여 CRISPR 시스템 기능을 향상시키는방법, 상기 특이적 sgRNA 및 Cas9 폴리펩타이드 또는 이를 코딩하는 폴리뉴클레오타이드를 포함하는 CRISPR 시스템, 상기 특이적 sgRNA 및 그의 이용에 관한 것이다. 본 발명에 따른 단일 가닥 가이드 RNA (sgRNA) 및 이를 이용한 CRISPR 시스템은 기존 sgRNA에 비하여 표적 DNA에 유의적으로 특이성 및 억제 효과를 향상시키는 바, 이러한 sgRNA와 이를 이용한 CRISPR 시스템은 유전자 가위를 이용한 유전자 교정용 조성물, 유전체 수준의 스크리닝, 암을 비롯한 다양한 질병의 치료제, 질병 진단 또는 이미징용 조성물 개발, 형질전환동물 개발 등의 폭넓은 분야에 이용될 수 있을 것으로 기대된다.
PCT/KR2018/015897 2017-12-14 2018-12-14 Crispr 시스템 기능 향상 방법 및 그의 이용 WO2019117660A2 (ko)

Applications Claiming Priority (2)

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KR10-2017-0172383 2017-12-14
KR20170172383 2017-12-14

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WO2019117660A2 WO2019117660A2 (ko) 2019-06-20
WO2019117660A3 true WO2019117660A3 (ko) 2019-08-08

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Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021521788A (ja) 2018-04-19 2021-08-30 ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア ゲノム編集のための組成物および方法
WO2021201653A1 (ko) * 2020-04-02 2021-10-07 중앙대학교 산학협력단 Crispr/cas9 시스템을 기반으로 한 유전체 편집 방법 및 이의 용도
WO2022124839A1 (ko) * 2020-12-09 2022-06-16 재단법인 아산사회복지재단 온-타겟 활성이 유지되고 오프-타겟 활성이 감소된 가이드 rna 및 이의 용도
CN116949039A (zh) * 2022-04-15 2023-10-27 西湖实验室(生命科学和生物医学浙江省实验室) 基于crispr的成像标记系统及其用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016094867A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2017106251A1 (en) * 2015-12-14 2017-06-22 President And Fellows Of Harvard College Cas discrimination using tuned guide rna
WO2017160752A1 (en) * 2016-03-14 2017-09-21 Intellia Therapeutics, Inc. Methods and compositions for gene editing
WO2017184786A1 (en) * 2016-04-19 2017-10-26 The Broad Institute Inc. Cpf1 complexes with reduced indel activity

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3230452A1 (en) * 2014-12-12 2017-10-18 The Broad Institute Inc. Dead guides for crispr transcription factors

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016094867A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2017106251A1 (en) * 2015-12-14 2017-06-22 President And Fellows Of Harvard College Cas discrimination using tuned guide rna
WO2017160752A1 (en) * 2016-03-14 2017-09-21 Intellia Therapeutics, Inc. Methods and compositions for gene editing
WO2017184786A1 (en) * 2016-04-19 2017-10-26 The Broad Institute Inc. Cpf1 complexes with reduced indel activity

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LIYANG, Z. ET AL.: "The effect of sequence mismatches on binding affinity and endonuclease activity are decoupled throughout the Cas9 binding site", BIORXIV, August 2017 (2017-08-01), XP055629593, Retrieved from the Internet <URL:https://doi.org/10.1101/176255> *

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KR20190071621A (ko) 2019-06-24
WO2019117660A2 (ko) 2019-06-20

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