JP2020507347A5 - - Google Patents

Download PDF

Info

Publication number
JP2020507347A5
JP2020507347A5 JP2019565062A JP2019565062A JP2020507347A5 JP 2020507347 A5 JP2020507347 A5 JP 2020507347A5 JP 2019565062 A JP2019565062 A JP 2019565062A JP 2019565062 A JP2019565062 A JP 2019565062A JP 2020507347 A5 JP2020507347 A5 JP 2020507347A5
Authority
JP
Japan
Prior art keywords
mutation
cas9 protein
modified cas9
modified
protein according
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2019565062A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020507347A (ja
JP7232531B2 (ja
Filing date
Publication date
Priority claimed from IT102017000016321A external-priority patent/IT201700016321A1/it
Application filed filed Critical
Publication of JP2020507347A publication Critical patent/JP2020507347A/ja
Publication of JP2020507347A5 publication Critical patent/JP2020507347A5/ja
Application granted granted Critical
Publication of JP7232531B2 publication Critical patent/JP7232531B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2019565062A 2017-02-14 2018-02-14 高忠実度cas9バリアントおよびそれらの利用 Active JP7232531B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
IT102017000016321 2017-02-14
IT102017000016321A IT201700016321A1 (it) 2017-02-14 2017-02-14 Mutanti di cas9 ad alta specificita' e loro applicazioni.
PCT/EP2018/053717 WO2018149888A1 (en) 2017-02-14 2018-02-14 High-fidelity cas9 variants and applications thereof

Publications (3)

Publication Number Publication Date
JP2020507347A JP2020507347A (ja) 2020-03-12
JP2020507347A5 true JP2020507347A5 (https=) 2021-05-06
JP7232531B2 JP7232531B2 (ja) 2023-03-03

Family

ID=59067822

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019565062A Active JP7232531B2 (ja) 2017-02-14 2018-02-14 高忠実度cas9バリアントおよびそれらの利用

Country Status (12)

Country Link
US (1) US11525127B2 (https=)
EP (1) EP3583209B8 (https=)
JP (1) JP7232531B2 (https=)
KR (1) KR20190116407A (https=)
CN (1) CN110520528B (https=)
AU (1) AU2018221644A1 (https=)
BR (1) BR112019016765A2 (https=)
CA (1) CA3053218A1 (https=)
EA (1) EA037825B1 (https=)
IT (1) IT201700016321A1 (https=)
MX (1) MX2019009651A (https=)
WO (1) WO2018149888A1 (https=)

Families Citing this family (66)

* 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
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
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
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CA2963820A1 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
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
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
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
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
US11377645B2 (en) 2016-12-16 2022-07-05 The Rockefeller University Compositions and methods using Cas9 with enhanced spacer acquisition function
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
IT201700016321A1 (it) 2017-02-14 2018-08-14 Univ Degli Studi Di Trento Mutanti di cas9 ad alta specificita' e loro applicazioni.
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
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
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
WO2018227114A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
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
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
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
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
KR20210060541A (ko) * 2018-09-19 2021-05-26 더 유니버시티 오브 홍콩 개선된 고처리량 조합 유전적 변형 시스템 및 최적화된 Cas9 효소 변이체
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160125A1 (en) * 2019-01-29 2020-08-06 Flagship Pioneering Innovations V, Inc. Compositions comprising an endonuclease and methods for purifying an endonuclease
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
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020257325A1 (en) 2019-06-17 2020-12-24 Vertex Pharmaceuticals Inc. Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20230174972A1 (en) * 2019-10-15 2023-06-08 Agency For Science, Technology And Research Assays For Measuring Nucleic Acid Modifying Enzyme Activity
WO2021122944A1 (en) 2019-12-18 2021-06-24 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
CA3163463A1 (en) * 2020-03-11 2021-09-16 Fuqiang Chen High fidelity spcas9 nucleases for genome modification
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
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
AU2021400745A1 (en) 2020-12-17 2023-07-20 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
CN112680430B (zh) * 2020-12-28 2023-06-06 南方医科大学 一种CRISPR SpCas9突变体及其应用
CN113005141A (zh) * 2021-01-05 2021-06-22 温州医科大学 高活性突变体构成的基因编辑工具及制备方法和修复先天性视网膜劈裂症致病基因的方法
CN112626070A (zh) * 2021-01-13 2021-04-09 海南微氪生物科技股份有限公司 一种基于切离酶基因编辑技术的定向修复系统
WO2022152746A1 (en) 2021-01-13 2022-07-21 Alia Therapeutics Srl K526d cas9 variants and applications thereof
WO2022238958A1 (en) 2021-05-12 2022-11-17 Crispr Therapeutics Ag Multiplex gene editing
WO2023285431A1 (en) 2021-07-12 2023-01-19 Alia Therapeutics Srl Compositions and methods for allele specific treatment of retinitis pigmentosa
EP4413146A2 (en) * 2021-10-05 2024-08-14 The Board of Trustees of the Leland Stanford Junior University Treatment of polycythemia vera via crispr/aav6 genome editing
CN120265763A (zh) * 2023-10-25 2025-07-04 上海津曼特生物科技有限公司 靶向ttr的基因编辑组合物
CN117887760A (zh) * 2024-01-22 2024-04-16 中国医学科学院北京协和医院 SQSTM1/p62基因新致病突变c.1143delC的敲入小鼠模型及其构建方法
CN118497204B (zh) * 2024-07-16 2024-12-03 西北农林科技大学深圳研究院 一种质粒CRISPR-pCas9n及基因编辑的方法与应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11685935B2 (en) * 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
WO2016123243A1 (en) * 2015-01-28 2016-08-04 The Regents Of The University Of California Methods and compositions for labeling a single-stranded target nucleic acid
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
US11214779B2 (en) * 2015-04-08 2022-01-04 University of Pittsburgh—of the Commonwealth System of Higher Education Activatable CRISPR/CAS9 for spatial and temporal control of genome editing
TWI813532B (zh) * 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的crispr酶突變
US9512446B1 (en) * 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
IT201700016321A1 (it) 2017-02-14 2018-08-14 Univ Degli Studi Di Trento Mutanti di cas9 ad alta specificita' e loro applicazioni.

Similar Documents

Publication Publication Date Title
JP2020507347A5 (https=)
JP2015517803A5 (https=)
RU2017134357A (ru) Сконструированный ботулинический нейротоксин
JP2004501618A5 (https=)
JP2008508859A5 (https=)
CN108949772A (zh) 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
JP2019520080A5 (https=)
DE69230671T2 (de) Impfstoffe gegen actinobacillus pleuropneumonie
JP2004531242A5 (https=)
TW214556B (https=)
JP2008545393A5 (https=)
CN105154436A (zh) 包含突变的核酸内切酶识别区dna及其基因组编辑应用
JPWO2021072298A5 (https=)
Corchero et al. Self-assembling, protein-based intracellular bacterial organelles: emerging vehicles for encapsulating, targeting and delivering therapeutical cargoes
ATE169958T1 (de) Herstellung der p1 proteine und impfstoffe gegen gonorrhöe
HRP20191535T1 (hr) Individualizirana cjepiva protiv raka
JPH05501956A (ja) 豚胸膜肺炎用ワクチン
CN120366260A (zh) Rna聚合酶变体及其应用
JP2020127419A5 (https=)
SE8105516L (sv) Syntetisk dna och forfarande for framstellning derav
US20210009641A1 (en) Expression of pneumococcal surface protein a (pspa)
CN109289046A (zh) 一种具核梭杆菌FomA蛋白质疫苗以及制备方法和应用
CN114438106A (zh) Rv2779c基因及其表达产物作为抗结核分枝杆菌靶点的应用
NZ505827A (en) Concatamers of non-identical genes for the same polypeptide and their application in vaccines
CN115074348B (zh) 一种广谱嵌合裂解酶ClyL、编码基因、重组载体、重组菌及其制备方法和应用