DK3277805T3 - CAS-9-retrovirale integrasesystemer til målrettet inkorporering af en dna-sekvens i en celles genom - Google Patents

CAS-9-retrovirale integrasesystemer til målrettet inkorporering af en dna-sekvens i en celles genom Download PDF

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DK3277805T3
DK3277805T3 DK16715977.1T DK16715977T DK3277805T3 DK 3277805 T3 DK3277805 T3 DK 3277805T3 DK 16715977 T DK16715977 T DK 16715977T DK 3277805 T3 DK3277805 T3 DK 3277805T3
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cas
genome
cell
dna sequence
retroviral integrase
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DK16715977.1T
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Ferrukh Sheikh
Tetsuya Kawamura
Gloria Mo
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Sohm Inc
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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DK16715977.1T 2015-03-31 2016-03-31 CAS-9-retrovirale integrasesystemer til målrettet inkorporering af en dna-sekvens i en celles genom DK3277805T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562140454P 2015-03-31 2015-03-31
US201562210451P 2015-08-27 2015-08-27
US201562240359P 2015-10-12 2015-10-12
PCT/US2016/025426 WO2016161207A1 (en) 2015-03-31 2016-03-31 Cas 9 retroviral integrase and cas 9 recombinase systems for targeted incorporation of a dna sequence into a genome of a cell or organism

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DK3277805T3 true DK3277805T3 (da) 2025-06-10

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US (2) US20180080051A1 (enExample)
EP (2) EP4600366A3 (enExample)
JP (4) JP2018513681A (enExample)
KR (3) KR102894634B1 (enExample)
CN (1) CN108124453B (enExample)
DK (1) DK3277805T3 (enExample)
ES (1) ES3030433T3 (enExample)
WO (1) WO2016161207A1 (enExample)

Families Citing this family (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 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
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
EP4600366A3 (en) * 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
US11911415B2 (en) 2015-06-09 2024-02-27 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for improving transplantation
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2017165826A1 (en) 2016-03-25 2017-09-28 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
GB201610041D0 (en) * 2016-06-08 2016-07-20 Oxford Genetics Ltd Methods
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US12188059B2 (en) 2016-09-13 2025-01-07 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
WO2018068053A2 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CA3044101A1 (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
US11278570B2 (en) 2016-12-16 2022-03-22 B-Mogen Biotechnologies, Inc. Enhanced hAT family transposon-mediated gene transfer and associated compositions, systems, and methods
CA3047313A1 (en) 2016-12-16 2018-06-21 B-Mogen Biotechnologies, Inc. Enhanced hat family transposon-mediated gene transfer and associated compositions, systems, and methods
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
WO2018129368A2 (en) 2017-01-06 2018-07-12 Editas Medicine, Inc. Methods of assessing nuclease cleavage
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include 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
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
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US11441135B2 (en) * 2017-07-07 2022-09-13 Toolgen Incorporated Target-specific CRISPR mutant
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base 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
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US20200255829A1 (en) * 2017-11-02 2020-08-13 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2019246486A2 (en) 2018-06-21 2019-12-26 B-Mogen Biotechnologies, Inc. ENHANCED hAT FAMILY TRANSPOSON-MEDIATED GENE TRANSFER AND ASSOCIATED COMPOSITIONS, SYSTEMS, AND METHODS
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
US20210363509A1 (en) * 2018-10-22 2021-11-25 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas9 Fusion Protein
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020131862A1 (en) * 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN113710693B (zh) 2019-02-25 2025-07-15 马萨诸塞大学 Dna结合结构域反式激活因子及其用途
WO2020181264A1 (en) * 2019-03-07 2020-09-10 The Trustees Of Columbia University In The City Of New York Rna-guided dna integration using tn7-like transposons
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12203070B2 (en) * 2019-05-23 2025-01-21 Christiana Care Gene Editing Institute, Inc. Gene knockout of NRF2 for treatment of cancer
JP7585237B2 (ja) * 2019-05-23 2024-11-18 クリスティアナ ケア ジーン エディティング インスティテュート，インコーポレーテッド がんを処置するためのバリアントｎｒｆ２の遺伝子ノックアウト
WO2020243085A1 (en) * 2019-05-24 2020-12-03 The Trustees Of Columbia University In The City Of New York Engineered cas-transposon system for programmable and site-directed dna transpositions
KR102990253B1 (ko) * 2019-06-11 2026-07-10 우니베르시타트 폼페우 파브라 표적화된 유전자 편집 작제물 및 이의 사용 방법
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20220403357A1 (en) * 2019-11-12 2022-12-22 The Broad Institute, Inc. Small type ii cas proteins and methods of use thereof
WO2021216503A1 (en) * 2020-04-20 2021-10-28 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022010241A1 (ko) * 2020-07-06 2022-01-13 한국과학기술연구원 질환 세포-특이적인 mirna에 의해 세포 생리 활성 조절 물질의 활성을 조절하는 복합체 및 이를 crispr/cas 시스템에 적용한 질환 특이적 유전자 조작용 복합체
WO2022007959A1 (zh) * 2020-07-10 2022-01-13 中国科学院动物研究所 一种编辑核酸的系统及方法
EP4185303A4 (en) * 2020-07-24 2024-10-30 University of Massachusetts Dna-binding domain transactivators and uses thereof
CN112159822A (zh) * 2020-09-30 2021-01-01 扬州大学 一种PS转座酶与CRISPR/dCpf1融合蛋白表达载体及其介导的定点整合方法
KR102803305B1 (ko) * 2020-11-25 2025-05-07 연세대학교 산학협력단 트랜스-스플라이싱 아데노-연관 바이러스 벡터를 포함하는 프라임에디팅용 조성물
CN116940673A (zh) * 2020-12-16 2023-10-24 庞培法布拉大学 可编程转座酶及其用途
AU2021403660A1 (en) * 2020-12-16 2023-07-06 Universitat Pompeu Fabra Programmable transposases and uses thereof
WO2022129430A1 (en) * 2020-12-16 2022-06-23 Universitat Pompeu Fabra Therapeutic lama2 payload for treatment of congenital muscular dystrophy
WO2024227131A1 (en) * 2023-04-27 2024-10-31 Rensselaer Polytechnic Institute Recombinant enzyme for the accurate insertion of dna sequences in eukaryotic cells
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
CN121915136A (zh) * 2024-11-22 2026-04-24 上海交通大学医学院附属第九人民医院 基于CRISPR/dCas融合蛋白的核酸原位检测系统及其应用

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997020038A1 (en) * 1995-12-01 1997-06-05 The Regents Of The University Of California Compositions and methods for site-directed integration into dna
US6080849A (en) 1997-09-10 2000-06-27 Vion Pharmaceuticals, Inc. Genetically modified tumor-targeted bacteria with reduced virulence
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US20040003420A1 (en) 2000-11-10 2004-01-01 Ralf Kuhn Modified recombinase
GB0400814D0 (en) * 2004-01-14 2004-02-18 Ark Therapeutics Ltd Integrating gene therapy vector
US20060252140A1 (en) * 2005-04-29 2006-11-09 Yant Stephen R Development of a transposon system for site-specific DNA integration in mammalian cells
US7951925B2 (en) 2006-05-25 2011-05-31 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
CA2669746C (en) 2006-12-14 2017-05-16 Dow Agrosciences Llc Optimized non-canonical zinc finger proteins
US8816153B2 (en) 2010-08-27 2014-08-26 Monsanto Technology Llc Recombinant DNA constructs employing site-specific recombination
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US9708589B2 (en) * 2012-12-18 2017-07-18 Monsanto Technology Llc Compositions and methods for custom site-specific DNA recombinases
WO2014134412A1 (en) 2013-03-01 2014-09-04 Regents Of The University Of Minnesota Talen-based gene correction
ES2749624T3 (es) 2013-03-14 2020-03-23 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
US10604771B2 (en) 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3004339B1 (en) * 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
JP6608807B2 (ja) * 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
US9322037B2 (en) * 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
CN104404036B (zh) * 2014-11-03 2017-12-01 赛业（苏州）生物科技有限公司 基于CRISPR/Cas9技术的条件性基因敲除方法
EP4600366A3 (en) * 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

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