WO2019066490A3 - 유전자 발현 조절을 위한 인위적인 게놈 조작 - Google Patents

유전자 발현 조절을 위한 인위적인 게놈 조작 Download PDF

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WO2019066490A3
WO2019066490A3 PCT/KR2018/011424 KR2018011424W WO2019066490A3 WO 2019066490 A3 WO2019066490 A3 WO 2019066490A3 KR 2018011424 W KR2018011424 W KR 2018011424W WO 2019066490 A3 WO2019066490 A3 WO 2019066490A3
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expression
gene expression
expression regulation
regulating
repeated genes
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PCT/KR2018/011424
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English (en)
French (fr)
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WO2019066490A2 (ko
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김석중
송동우
이재영
이정민
조규본
배희숙
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주식회사 툴젠
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Priority to CA3077153A priority Critical patent/CA3077153A1/en
Application filed by 주식회사 툴젠 filed Critical 주식회사 툴젠
Priority to CN201880076752.2A priority patent/CN111527208B/zh
Priority to RU2020114785A priority patent/RU2767201C2/ru
Priority to EP18862957.0A priority patent/EP3690047A4/en
Priority to SG11202002130WA priority patent/SG11202002130WA/en
Priority to AU2018339164A priority patent/AU2018339164B2/en
Priority to BR112020006428-9A priority patent/BR112020006428A2/pt
Priority to JP2020513755A priority patent/JP2020536501A/ja
Publication of WO2019066490A2 publication Critical patent/WO2019066490A2/ko
Publication of WO2019066490A3 publication Critical patent/WO2019066490A3/ko
Priority to US16/776,707 priority patent/US11572574B2/en
Priority to JP2021170086A priority patent/JP7440043B2/ja
Priority to US18/147,156 priority patent/US20230295652A1/en

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Abstract

본 발명은 중복 유전자의 발현을 조절하기 위한 발현 조절용 조성물 또는 이를 이용한 방법에 관한 것이다. 보다 구체적으로, 중복 유전자의 전사 조절 영역을 표적화할 수 있는 가이드핵산을 포함하는 발현 조절용 조성물 및 이를 이용한 중복 유전자의 전사 조절 영역을 인위적으로 조작 및/또는 변형시켜 중복 유전자의 발현을 조절하는 방법에 관한 것이다. 또한 중복 유전자의 발현을 조절하기 위한 발현 조절용 조성물을 이용하여 유전자 중복에 의해 발생하는 질환을 치료 또는 개선하는 방법에 관한 것이다.
PCT/KR2018/011424 2017-09-28 2018-09-27 유전자 발현 조절을 위한 인위적인 게놈 조작 WO2019066490A2 (ko)

Priority Applications (11)

Application Number Priority Date Filing Date Title
AU2018339164A AU2018339164B2 (en) 2017-09-28 2018-09-27 Artificial genome manipulation for gene expression regulation
CN201880076752.2A CN111527208B (zh) 2017-09-28 2018-09-27 用于基因表达调节的人工基因组操纵
RU2020114785A RU2767201C2 (ru) 2017-09-28 2018-09-27 Искусственная модификация генома для регуляции экспрессии гена
EP18862957.0A EP3690047A4 (en) 2017-09-28 2018-09-27 ARTIFICIAL GENOME MANIPULATION FOR GENE EXPRESSION REGULATION
SG11202002130WA SG11202002130WA (en) 2017-09-28 2018-09-27 Artificial genome manipulation for gene expression regulation
CA3077153A CA3077153A1 (en) 2017-09-28 2018-09-27 Artificial genome manipulation for gene expression regulation
BR112020006428-9A BR112020006428A2 (pt) 2017-09-28 2018-09-27 manipulação de genoma artificial para regulação de expressão de genes
JP2020513755A JP2020536501A (ja) 2017-09-28 2018-09-27 遺伝子発現調節のための人為的なゲノム操作
US16/776,707 US11572574B2 (en) 2017-09-28 2020-01-30 Artificial genome manipulation for gene expression regulation
JP2021170086A JP7440043B2 (ja) 2017-09-28 2021-10-18 遺伝子発現調節のための人為的なゲノム操作
US18/147,156 US20230295652A1 (en) 2017-09-28 2022-12-28 Artificial genome manipulation for gene expression regulation

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762564478P 2017-09-28 2017-09-28
US62/564,478 2017-09-28
US201762565868P 2017-09-29 2017-09-29
US62/565,868 2017-09-29

Related Child Applications (1)

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US16/776,707 Continuation-In-Part US11572574B2 (en) 2017-09-28 2020-01-30 Artificial genome manipulation for gene expression regulation

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WO2019066490A2 WO2019066490A2 (ko) 2019-04-04
WO2019066490A3 true WO2019066490A3 (ko) 2019-06-27

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PCT/KR2018/011424 WO2019066490A2 (ko) 2017-09-28 2018-09-27 유전자 발현 조절을 위한 인위적인 게놈 조작

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EP (1) EP3690047A4 (ko)
JP (2) JP2020536501A (ko)
CN (1) CN111527208B (ko)
AU (1) AU2018339164B2 (ko)
BR (1) BR112020006428A2 (ko)
CA (1) CA3077153A1 (ko)
RU (2) RU2767201C2 (ko)
SG (1) SG11202002130WA (ko)
WO (1) WO2019066490A2 (ko)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110577970B (zh) * 2019-08-08 2022-11-18 复旦大学 CRISPR/Sa-SlutCas9基因编辑系统及其应用
CN110577969B (zh) * 2019-08-08 2022-07-22 复旦大学 CRISPR/Sa-SlugCas9基因编辑系统及其应用
CN110577972B (zh) * 2019-08-08 2022-10-11 复旦大学 CRISPR/Sa-ShaCas9基因编辑系统及其应用
KR20230134477A (ko) * 2020-12-01 2023-09-21 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 말초 미엘린 단백질-22의 발현 억제를 위한 생성물및 방법
WO2022167421A1 (en) * 2021-02-02 2022-08-11 Limagrain Europe Linkage of a distal promoter to a gene of interest by gene editing to modify gene expression
CN114426982B (zh) * 2022-02-22 2024-03-22 上海交通大学 一种细胞代谢重编程的方法及其应用
CN116536357A (zh) * 2023-04-17 2023-08-04 中国医学科学院输血研究所 构建CRISPR/Cas12a中sgRNA剪切活性筛选系统的方法

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20150101446A (ko) * 2012-10-23 2015-09-03 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
KR20150105633A (ko) * 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 시스템, 방법 및 최적화된 가이드 조성물의 조작
KR20160050069A (ko) * 2013-09-06 2016-05-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Cas9 변이체 및 그의 용도
WO2017035416A2 (en) * 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
WO2017083852A1 (en) * 2015-11-13 2017-05-18 MOORE, Tara Methods for the treatment of corneal dystrophies

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3456831T (pt) * 2013-04-16 2021-09-10 Regeneron Pharma Modificação alvejada do genoma de rato
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
CN111269902A (zh) * 2013-12-12 2020-06-12 布罗德研究所有限公司 Crispr-cas系统和组合物的递送及用途
CN110248957B (zh) * 2016-11-14 2023-12-19 株式会社图尔金 经人工操纵的sc功能控制系统
CN110249051A (zh) * 2016-12-08 2019-09-17 凯斯西储大学 增强功能性髓鞘产生的方法和组合物

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20150101446A (ko) * 2012-10-23 2015-09-03 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
KR20150105633A (ko) * 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 시스템, 방법 및 최적화된 가이드 조성물의 조작
KR20160050069A (ko) * 2013-09-06 2016-05-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Cas9 변이체 및 그의 용도
WO2017035416A2 (en) * 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
WO2017083852A1 (en) * 2015-11-13 2017-05-18 MOORE, Tara Methods for the treatment of corneal dystrophies

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JP7440043B2 (ja) 2024-02-28
BR112020006428A2 (pt) 2020-09-24
EP3690047A2 (en) 2020-08-05
SG11202002130WA (en) 2020-04-29
EP3690047A4 (en) 2021-11-03
AU2018339164A1 (en) 2020-04-09
RU2767201C2 (ru) 2022-03-16
CN111527208B (zh) 2024-05-14
WO2019066490A2 (ko) 2019-04-04
KR20190037145A (ko) 2019-04-05
RU2020114785A3 (ko) 2021-10-28
CN111527208A (zh) 2020-08-11
RU2020114785A (ru) 2021-10-28
CA3077153A1 (en) 2019-04-04
JP2020536501A (ja) 2020-12-17
JP2022023144A (ja) 2022-02-07
AU2018339164B2 (en) 2022-06-16
RU2022103641A (ru) 2022-03-18

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