EA037825B1 - ВЫСОКОТОЧНЫЕ ВАРИАНТЫ Cas9 И ИХ ПРИМЕНЕНИЯ - Google Patents
ВЫСОКОТОЧНЫЕ ВАРИАНТЫ Cas9 И ИХ ПРИМЕНЕНИЯ Download PDFInfo
- Publication number
- EA037825B1 EA037825B1 EA201991913A EA201991913A EA037825B1 EA 037825 B1 EA037825 B1 EA 037825B1 EA 201991913 A EA201991913 A EA 201991913A EA 201991913 A EA201991913 A EA 201991913A EA 037825 B1 EA037825 B1 EA 037825B1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- mutations
- cas9
- seq
- target
- spcas9
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 (2)
| Publication Number | Publication Date |
|---|---|
| EA201991913A1 EA201991913A1 (ru) | 2020-01-22 |
| EA037825B1 true EA037825B1 (ru) | 2021-05-25 |
Family
ID=59067822
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA201991913A EA037825B1 (ru) | 2017-02-14 | 2018-02-14 | ВЫСОКОТОЧНЫЕ ВАРИАНТЫ Cas9 И ИХ ПРИМЕНЕНИЯ |
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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| EP4464338A3 (en) | 2014-11-07 | 2025-02-12 | Editas Medicine, Inc. | Systems for improving crispr/cas-mediated genome-editing |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| CN108026526B (zh) | 2015-06-09 | 2023-05-12 | 爱迪塔斯医药公司 | 用于改善移植的crispr/cas相关方法和组合物 |
| EP3353296B1 (en) | 2015-09-24 | 2020-11-04 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| US11597924B2 (en) | 2016-03-25 | 2023-03-07 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| US11236313B2 (en) | 2016-04-13 | 2022-02-01 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | 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 |
| KR20260019012A (ko) | 2016-10-07 | 2026-02-09 | 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. | S. 피오게네스 cas9 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드 |
| US11242542B2 (en) * | 2016-10-07 | 2022-02-08 | Integrated Dna Technologies, Inc. | S. pyogenes Cas9 mutant genes and polypeptides encoded by same |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| US11377645B2 (en) | 2016-12-16 | 2022-07-05 | The Rockefeller University | Compositions and methods using Cas9 with enhanced spacer acquisition function |
| EP3555297A1 (en) | 2016-12-19 | 2019-10-23 | 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 |
| JP7229923B2 (ja) | 2017-01-06 | 2023-02-28 | エディタス・メディシン、インコーポレイテッド | ヌクレアーゼ切断を評価する方法 |
| IT201700016321A1 (it) | 2017-02-14 | 2018-08-14 | Univ Degli Studi Di Trento | Mutanti di cas9 ad alta specificita' e loro applicazioni. |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | Editas Medicine, Inc. | Methods and systems for analyzing guide RNA molecules |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| MX2019014640A (es) | 2017-06-09 | 2020-10-05 | Editas Medicine Inc | Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas. |
| 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 |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| 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 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | 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 |
| EP4253549A3 (en) * | 2018-09-19 | 2023-12-06 | The University of Hong Kong | Improved high-throughput combinatorial genetic modification system and optimized cas9 enzyme variants |
| 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 |
| US20220249625A1 (en) * | 2019-01-29 | 2022-08-11 | Flagship Pioneering Innovations V, Inc. | Compositions comprising an endonuclease and methods for purifying an endonuclease |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP3983542A2 (en) | 2019-06-17 | 2022-04-20 | CRISPR Therapeutics AG | Methods and compositions for improved homology directed repair |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| JP7680440B2 (ja) * | 2019-10-15 | 2025-05-20 | エージェンシー フォー サイエンス, テクノロジー アンド リサーチ | 核酸修飾酵素活性を測定するためのアッセイ法 |
| WO2021122944A1 (en) | 2019-12-18 | 2021-06-24 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| US20230058352A1 (en) * | 2020-03-11 | 2023-02-23 | Sigma-Aldrich Co. Llc | High Fidelity SpCas9 Nucleases for Genome Modification |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| WO2022018638A1 (en) | 2020-07-21 | 2022-01-27 | Crispr Therapeutics Ag | Genome-editing compositions and methods to modulate faah for treatment of neurological disorders |
| WO2022133246A1 (en) | 2020-12-17 | 2022-06-23 | 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 |
| WO2022152746A1 (en) | 2021-01-13 | 2022-07-21 | Alia Therapeutics Srl | K526d cas9 variants and applications thereof |
| 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 |
| 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 |
| CN120225668A (zh) * | 2023-10-25 | 2025-06-27 | 石药集团中奇制药技术(石家庄)有限公司 | 一种高效低脱靶的基因编辑工具 |
| 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 |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016164797A1 (en) * | 2015-04-08 | 2016-10-13 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Activatable crispr/cas9 for spatial and temporal control of genome editing |
| US9512446B1 (en) * | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| WO2016205613A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzyme mutations reducing off-target effects |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3004339B1 (en) * | 2013-05-29 | 2021-07-07 | Cellectis | New compact scaffold of cas9 in the type ii crispr system |
| EP3250689B1 (en) * | 2015-01-28 | 2020-11-04 | The Regents of The University of California | Methods and compositions for labeling a single-stranded target nucleic acid |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| IT201700016321A1 (it) | 2017-02-14 | 2018-08-14 | Univ Degli Studi Di Trento | Mutanti di cas9 ad alta specificita' e loro applicazioni. |
-
2017
- 2017-02-14 IT IT102017000016321A patent/IT201700016321A1/it unknown
-
2018
- 2018-02-14 EA EA201991913A patent/EA037825B1/ru unknown
- 2018-02-14 WO PCT/EP2018/053717 patent/WO2018149888A1/en not_active Ceased
- 2018-02-14 MX MX2019009651A patent/MX2019009651A/es unknown
- 2018-02-14 CA CA3053218A patent/CA3053218A1/en active Pending
- 2018-02-14 US US16/485,253 patent/US11525127B2/en active Active
- 2018-02-14 AU AU2018221644A patent/AU2018221644A1/en not_active Abandoned
- 2018-02-14 JP JP2019565062A patent/JP7232531B2/ja active Active
- 2018-02-14 EP EP18708920.6A patent/EP3583209B8/en active Active
- 2018-02-14 CN CN201880011895.5A patent/CN110520528B/zh active Active
- 2018-02-14 BR BR112019016765-0A patent/BR112019016765A2/pt not_active IP Right Cessation
- 2018-02-14 KR KR1020197026441A patent/KR20190116407A/ko not_active Abandoned
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016164797A1 (en) * | 2015-04-08 | 2016-10-13 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Activatable crispr/cas9 for spatial and temporal control of genome editing |
| WO2016205613A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzyme mutations reducing off-target effects |
| US9512446B1 (en) * | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
Non-Patent Citations (4)
| Title |
|---|
| BENJAMIN P. KLEINSTIVER, PATTANAYAK VIKRAM, PREW MICHELLE S., TSAI SHENGDAR Q., NGUYEN NHU T., ZHENG ZONGLI, JOUNG J. KEITH: "High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects", NATURE, MACMILLAN JOURNALS LTD., ETC, LONDON, vol. 529, no. 7587, 28 January 2016 (2016-01-28), London, pages 490 - 495, XP055303390, ISSN: 0028-0836, DOI: 10.1038/nature16526 * |
| CASINI A. ET AL.: "Identification of high-fidelity Cas9 variants using a yeast-based screening", HUMAN GENE THERAPY, vol. 27, no. 11, 18 October 2016 (2016-10-18), 21 October 2016 (2016-10-21), page A41, & CONFERENCE ON CHANGING THE FACE OF MODERN MEDICINE - STEM CELLS AND GENE THERAPY; FLORENCE, ITALY, the whole document * |
| IAN M. SLAYMAKER, LINYI GAO, BERND ZETSCHE, DAVID A. SCOTT, WINSTON X. YAN, FENG ZHANG: "Rationally engineered Cas9 nucleases with improved specificity", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, US, vol. 351, no. 6268, 1 January 2016 (2016-01-01), US, pages 84 - 88, XP002757561, ISSN: 0036-8075, DOI: 10.1126/science.aad5227 * |
| M. JINEK, F. JIANG, D. W. TAYLOR, S. H. STERNBERG, E. KAYA, E. MA, C. ANDERS, M. HAUER, K. ZHOU, S. LIN, M. KAPLAN, A. T. IAVARONE: "Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, vol. 343, no. 6176, 14 March 2014 (2014-03-14), pages 1247997 - 1247997, XP055149157, ISSN: 00368075, DOI: 10.1126/science.1247997 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP7232531B2 (ja) | 2023-03-03 |
| CN110520528B (zh) | 2024-08-13 |
| EP3583209B8 (en) | 2025-02-26 |
| WO2018149888A1 (en) | 2018-08-23 |
| MX2019009651A (es) | 2019-12-18 |
| CA3053218A1 (en) | 2018-08-23 |
| US11525127B2 (en) | 2022-12-13 |
| EP3583209C0 (en) | 2024-12-04 |
| IT201700016321A1 (it) | 2018-08-14 |
| EP3583209A1 (en) | 2019-12-25 |
| BR112019016765A2 (pt) | 2020-05-26 |
| EP3583209B1 (en) | 2024-12-04 |
| EA201991913A1 (ru) | 2020-01-22 |
| AU2018221644A1 (en) | 2019-08-29 |
| JP2020507347A (ja) | 2020-03-12 |
| KR20190116407A (ko) | 2019-10-14 |
| CN110520528A (zh) | 2019-11-29 |
| US20200149020A1 (en) | 2020-05-14 |
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