JP2017538427A - Crispr系組成物及び使用方法 - Google Patents

Crispr系組成物及び使用方法 Download PDF

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JP2017538427A
JP2017538427A JP2017532803A JP2017532803A JP2017538427A JP 2017538427 A JP2017538427 A JP 2017538427A JP 2017532803 A JP2017532803 A JP 2017532803A JP 2017532803 A JP2017532803 A JP 2017532803A JP 2017538427 A JP2017538427 A JP 2017538427A
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tracrrna
crrna
seq
rna
isolated
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コーリングウッド,マイケル・アレン
ジャコビ,アシュリー・メイ
レッティグ,ギャレット・リチャード
シューバート,モリー・スー
ベールケ,マーク・アーロン
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インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド
インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド
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Publication of JP2017538427A publication Critical patent/JP2017538427A/ja
Priority to JP2021071664A priority Critical patent/JP2021118714A/ja
Priority to JP2023115668A priority patent/JP7770361B2/ja
Priority to JP2025132121A priority patent/JP2025160487A/ja
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JP2017532803A 2014-12-18 2015-12-18 Crispr系組成物及び使用方法 Pending JP2017538427A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021071664A JP2021118714A (ja) 2014-12-18 2021-04-21 Crispr系組成物及び使用方法
JP2023115668A JP7770361B2 (ja) 2014-12-18 2023-07-14 Crispr系組成物及び使用方法
JP2025132121A JP2025160487A (ja) 2014-12-18 2025-08-07 Crispr系組成物及び使用方法

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Application Number Priority Date Filing Date Title
US201462093588P 2014-12-18 2014-12-18
US62/093,588 2014-12-18
US201562239546P 2015-10-09 2015-10-09
US62/239,546 2015-10-09
PCT/US2015/066942 WO2016100951A2 (en) 2014-12-18 2015-12-18 Crispr-based compositions and methods of use

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JP2017532803A Pending JP2017538427A (ja) 2014-12-18 2015-12-18 Crispr系組成物及び使用方法
JP2021071664A Pending JP2021118714A (ja) 2014-12-18 2021-04-21 Crispr系組成物及び使用方法
JP2023115668A Active JP7770361B2 (ja) 2014-12-18 2023-07-14 Crispr系組成物及び使用方法
JP2025132121A Pending JP2025160487A (ja) 2014-12-18 2025-08-07 Crispr系組成物及び使用方法

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JP2023115668A Active JP7770361B2 (ja) 2014-12-18 2023-07-14 Crispr系組成物及び使用方法
JP2025132121A Pending JP2025160487A (ja) 2014-12-18 2025-08-07 Crispr系組成物及び使用方法

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US (4) US9840702B2 (https=)
EP (2) EP3234133B1 (https=)
JP (4) JP2017538427A (https=)
CN (3) CN112877327B (https=)
AU (3) AU2015364282B2 (https=)
CA (1) CA2970683A1 (https=)
DK (1) DK3234133T3 (https=)
WO (1) WO2016100951A2 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019156475A1 (ko) * 2018-02-06 2019-08-15 주식회사 진씨커 돌연변이 세포 유리 유전자 분리 키트 및 이를 이용한 돌연변이 세포 유리 유전자 분리 방법
CN111065747A (zh) * 2018-02-06 2020-04-24 株式会社金希科 突变细胞游离基因分离试剂盒及利用其的突变细胞游离基因分离方法
JP2021526804A (ja) * 2018-06-08 2021-10-11 インテリア セラピューティクス,インコーポレイテッド 遺伝子編集のための修飾されたガイドrna
JP2023526057A (ja) * 2020-05-12 2023-06-20 ユニバーシティー オブ マサチューセッツ Crisprゲノム編集のための修飾されたガイドrna

Families Citing this family (79)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US10040048B1 (en) 2014-09-25 2018-08-07 Synthego Corporation Automated modular system and method for production of biopolymers
CN112877327B (zh) 2014-12-18 2024-11-22 综合基因技术公司 基于crispr的组合物和使用方法
EP3280803B1 (en) 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
EP4545544A3 (en) 2015-06-29 2025-10-08 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
US11279928B2 (en) 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
IL241462A0 (en) 2015-09-10 2015-11-30 Yeda Res & Dev Heterologous engineering of betalain pigments in plants
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したCas9蛋白質
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
US11845933B2 (en) 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
US10767175B2 (en) * 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
IL247752A0 (en) 2016-09-11 2016-11-30 Yeda Res & Dev Compositions and methods for modulating gene expression for site-directed mutagenesis
CA3034931A1 (en) 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
DE102016118109A1 (de) * 2016-09-26 2018-03-29 Newfrey Llc Fügeverfahren zum vorlochfreien Verbinden von wenigstens einem ersten Bauteil mit einem zweiten Bauteil
CN118726313A (zh) 2016-10-07 2024-10-01 综合Dna技术公司 化脓链球菌cas9突变基因和由其编码的多肽
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018098383A1 (en) 2016-11-22 2018-05-31 Integrated Dna Technologies, Inc. Crispr/cpf1 systems and methods
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
WO2018111946A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing detection
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018175636A2 (en) * 2017-03-22 2018-09-27 Novartis Ag Compositions and methods for immunooncology
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
JP2020515258A (ja) * 2017-03-31 2020-05-28 アジェノビア コーポレーション 抗ウイルス治療剤
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
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
WO2019011118A1 (zh) 2017-07-14 2019-01-17 苏州克睿基因生物科技有限公司 一种基因编辑系统及基因编辑的方法
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US20190076814A1 (en) * 2017-09-11 2019-03-14 Synthego Corporation Biopolymer synthesis system and method
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US20200354413A1 (en) 2017-12-15 2020-11-12 Dana-Farber Cancer Institute, Inc. Stabilized peptide-mediated targeted protein degradation
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019126037A1 (en) * 2017-12-19 2019-06-27 City Of Hope Modified tracrrnas grnas, and uses thereof
WO2019147275A1 (en) * 2018-01-26 2019-08-01 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
WO2019147743A1 (en) * 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
CN109504711A (zh) * 2018-02-14 2019-03-22 复旦大学 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性基因组位点相互作用DNA的方法
WO2019183000A1 (en) * 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
CN112105732A (zh) * 2018-05-10 2020-12-18 先正达参股股份有限公司 用于多核苷酸的靶向编辑的方法和组合物
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
CN110616187B (zh) * 2018-06-20 2021-12-03 西安桑尼赛尔生物医药有限公司 CRISPR-Cas9高效敲入嵌合抗原受体基因到T细胞特定基因组位点的方法及应用
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
IL325857A (en) 2018-08-08 2026-03-01 Integrated Dna Tech Inc Novel mutations that enhance DNA cleavage activity of ACIDAMINOCOCCUS SP. CPF1
EP3617325B1 (en) 2018-08-29 2021-10-20 Leibniz-Institut für Pflanzengenetik und Kulturpflanzenforschung (IPK) Rna-guided endonuclease dna labeling
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN109660518B (zh) * 2018-11-22 2020-12-18 北京六方云信息技术有限公司 网络的通信数据检测方法、装置以及机器可读存储介质
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020226864B2 (en) 2019-02-22 2023-09-28 Integrated Dna Technologies, Inc. Lachnospiraceae Bacterium ND2006 Cas12a mutant genes and polypeptides encoded by same
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
WO2020208191A1 (en) 2019-04-10 2020-10-15 Technische Universität Hamburg Novel dhap synthase
LU101172B1 (en) 2019-04-10 2020-10-12 Technische Univ Hamburg Method for in vivo screening of engineered enzyme variants
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220403553A1 (en) 2019-09-12 2022-12-22 Glaxosmithkline Intellectual Property Development Limited Method for screening libraries
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4438729A3 (en) * 2019-12-09 2025-03-26 Caribou Biosciences, Inc. Crispr abasic restricted nucleotides and crispr accuracy via analogs
US12012433B1 (en) 2020-02-27 2024-06-18 Integrated Dna Technologies Inc. Expression and purification of Cas enzymes
CN116096878A (zh) 2020-05-01 2023-05-09 合成Dna技术公司 在非经典tttt前间区序列邻近基序处具有增强的切割活性的毛螺菌科菌种cas12a突变体
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
TW202424186A (zh) * 2022-08-25 2024-06-16 美商生命編輯治療學公司 Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾
US20260071238A1 (en) 2022-09-02 2026-03-12 Janssen Biotech, Inc. Materials and processes for engineering hypoimmunogenicity
CN121241136A (zh) * 2023-04-07 2025-12-30 基因泰克公司 经修饰的指导rna
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014065596A1 (en) * 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014124226A1 (en) * 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
US20140273232A1 (en) * 2012-12-12 2014-09-18 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014144592A2 (en) * 2013-03-15 2014-09-18 The General Hospital Corporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
HK1199066A1 (en) 2011-08-18 2015-06-19 Nestec S.A. Compositions and methods for detecting allelic variants
US9895451B2 (en) * 2011-12-02 2018-02-20 Yale University Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP3363902B1 (en) * 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2894684A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
US20140212869A1 (en) * 2013-01-25 2014-07-31 Agilent Technologies, Inc. Nucleic Acid Proximity Assay Involving the Formation of a Three-way junction
WO2014131833A1 (en) * 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
MX374090B (es) * 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
WO2014194190A1 (en) * 2013-05-30 2014-12-04 The Penn State Research Foundation Gene targeting and genetic modification of plants via rna-guided genome editing
CA3109801C (en) 2013-08-22 2024-01-09 Andrew Cigan Plant genome modification using guide rna/cas endonuclease systems and methods of use
JP6361121B2 (ja) 2013-11-29 2018-07-25 三菱ケミカル株式会社 粘着剤樹脂組成物
US20150376586A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
WO2016080097A1 (ja) * 2014-11-17 2016-05-26 国立大学法人東京医科歯科大学 簡便で高効率の遺伝子改変非ヒト哺乳動物の作製方法
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
CN112877327B (zh) 2014-12-18 2024-11-22 综合基因技术公司 基于crispr的组合物和使用方法
EP3280803B1 (en) 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2017053431A2 (en) 2015-09-21 2017-03-30 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
WO2019147275A1 (en) 2018-01-26 2019-08-01 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014065596A1 (en) * 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
US20140273232A1 (en) * 2012-12-12 2014-09-18 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014124226A1 (en) * 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
WO2014144592A2 (en) * 2013-03-15 2014-09-18 The General Hospital Corporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
PLOS ONE, 2014, VOL. 9, ISSUE 10, E109213, JPN6020000545, ISSN: 0004410812 *
SCIENCE, 2012, VOL. 337, PP. 816-821 AND SUPPLEMENTARY MATERIALS, JPN6020000543, ISSN: 0004410811 *

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019156475A1 (ko) * 2018-02-06 2019-08-15 주식회사 진씨커 돌연변이 세포 유리 유전자 분리 키트 및 이를 이용한 돌연변이 세포 유리 유전자 분리 방법
CN111065747A (zh) * 2018-02-06 2020-04-24 株式会社金希科 突变细胞游离基因分离试剂盒及利用其的突变细胞游离基因分离方法
US11613776B2 (en) 2018-02-06 2023-03-28 Genecker Co., Ltd. Mutant cell-free DNA isolation kit and mutant cell-free DNA isolation method using the same
CN111065747B (zh) * 2018-02-06 2023-10-31 株式会社金希科 突变细胞游离基因分离试剂盒及利用其的突变细胞游离基因分离方法
JP2021526804A (ja) * 2018-06-08 2021-10-11 インテリア セラピューティクス,インコーポレイテッド 遺伝子編集のための修飾されたガイドrna
JP7832761B2 (ja) 2018-06-08 2026-03-18 インテリア セラピューティクス,インコーポレイテッド 遺伝子編集のための修飾されたガイドrna
JP2023526057A (ja) * 2020-05-12 2023-06-20 ユニバーシティー オブ マサチューセッツ Crisprゲノム編集のための修飾されたガイドrna

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