CN111527204B - 靶基因改造用组合物 - Google Patents

靶基因改造用组合物 Download PDF

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Publication number
CN111527204B
CN111527204B CN201880084068.9A CN201880084068A CN111527204B CN 111527204 B CN111527204 B CN 111527204B CN 201880084068 A CN201880084068 A CN 201880084068A CN 111527204 B CN111527204 B CN 111527204B
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rna
seq
composition
nucleic acid
cas9
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CN111527204A (zh
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堀田秋津
井福正隆
藤本直子
岩渕久美子
见城江利也
莳田幸正
落合留美子
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Kyoto University NUC
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Takeda Pharmaceutical Co Ltd
Kyoto University NUC
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    • C12N15/90Stable introduction of foreign DNA into chromosome
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CN201880084068.9A 2017-12-28 2018-12-27 靶基因改造用组合物 Active CN111527204B (zh)

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JP2017254798 2017-12-28
JP2017-254798 2017-12-28
PCT/JP2018/048034 WO2019131829A1 (ja) 2017-12-28 2018-12-27 標的遺伝子改変用組成物

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CN111527204B true CN111527204B (zh) 2023-09-01

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US (1) US12031130B2 (https=)
EP (1) EP3733844B1 (https=)
JP (1) JP7288282B2 (https=)
KR (1) KR102799255B1 (https=)
CN (1) CN111527204B (https=)
AU (1) AU2018397951B2 (https=)
CA (1) CA3086885A1 (https=)
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Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
US20200331841A1 (en) 2017-12-28 2020-10-22 Takeda Pharmaceutical Company Limited Cationic lipids
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
EP3842538A4 (en) * 2018-08-10 2022-06-08 Kyoto University METHOD OF TRANSFECTION INTO CARDIOMYOCYTES USING CATIONIC LIPID
US12479791B2 (en) 2018-08-10 2025-11-25 Takeda Pharmaceutical Company Limited Cationic lipid
TW202208626A (zh) * 2020-04-24 2022-03-01 美商生命編輯公司 Rna引導核酸酶及其活性片段與變體,以及使用方法
EP4225907A4 (en) * 2020-10-12 2025-01-22 Duke University CRISPR/CAS-BASED BASE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
CN113528437B (zh) * 2021-07-07 2023-08-25 中国医学科学院血液病医院(中国医学科学院血液学研究所) 一种增强基因编辑效率的试剂盒及其应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104116643A (zh) * 2014-07-10 2014-10-29 上海应用技术学院 一种包覆曲酸双棕榈酸酯的纳米固体脂质载体及制备方法
WO2016197133A1 (en) * 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
WO2017072590A1 (en) * 2015-10-28 2017-05-04 Crispr Therapeutics Ag Materials and methods for treatment of duchenne muscular dystrophy

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7223887B2 (en) 2001-12-18 2007-05-29 The University Of British Columbia Multivalent cationic lipids and methods of using same in the production of lipid particles
AU2003240978A1 (en) 2002-05-31 2003-12-19 Genteric, Inc. Multi-functional polyamines for delivery of biologically-active polynucleotides
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
KR101773643B1 (ko) 2010-04-28 2017-08-31 교와 핫꼬 기린 가부시키가이샤 양이온성 지질
WO2012028524A2 (en) 2010-08-30 2012-03-08 F. Hoffmann-La Roche Ag Method for producing a lipid particle, the lipid particle itself and its use
JP6108197B2 (ja) 2012-07-02 2017-04-05 日油株式会社 三級アミノ基含有脂質の製造方法
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
EP3093283B1 (en) 2014-01-09 2018-12-19 Eisai R&D Management Co., Ltd. Cationic lipid
US20170015994A1 (en) * 2014-02-24 2017-01-19 Massachusetts Institute Of Technology Methods for in vivo genome editing
KR101882634B1 (ko) 2014-08-07 2018-07-26 다케다 야쿠힝 고교 가부시키가이샤 양이온성 지질
CN106714845A (zh) 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
EP3275448B1 (en) 2015-03-24 2025-10-29 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticles
CN104873976B (zh) 2015-05-08 2016-04-20 厦门成坤生物技术有限公司 一类阳离子类脂化合物的应用
TWI894123B (zh) 2017-12-27 2025-08-21 日商武田藥品工業有限公司 含核酸之脂質奈米粒子及其用途

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
CN104116643A (zh) * 2014-07-10 2014-10-29 上海应用技术学院 一种包覆曲酸双棕榈酸酯的纳米固体脂质载体及制备方法
WO2016197133A1 (en) * 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
WO2017072590A1 (en) * 2015-10-28 2017-05-04 Crispr Therapeutics Ag Materials and methods for treatment of duchenne muscular dystrophy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo;Hao Yin et al.;Nat Biotechnol;第34卷(第34期);摘要,图1-3 *

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WO2019131829A1 (ja) 2019-07-04
CN111527204A (zh) 2020-08-11
AU2018397951B2 (en) 2025-06-26
US20210371854A1 (en) 2021-12-02
TW201938202A (zh) 2019-10-01
KR20200104318A (ko) 2020-09-03
EP3733844A4 (en) 2021-11-03
AU2018397951A1 (en) 2020-07-09
JP7288282B2 (ja) 2023-06-07
EP3733844A1 (en) 2020-11-04
TWI825057B (zh) 2023-12-11
US12031130B2 (en) 2024-07-09
CA3086885A1 (en) 2019-07-04
JPWO2019131829A1 (ja) 2021-02-04
EP3733844B1 (en) 2025-04-02
KR102799255B1 (ko) 2025-04-24

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