KR20250171479A - 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템 - Google Patents

세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템

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KR20250171479A
KR20250171479A KR1020257040002A KR20257040002A KR20250171479A KR 20250171479 A KR20250171479 A KR 20250171479A KR 1020257040002 A KR1020257040002 A KR 1020257040002A KR 20257040002 A KR20257040002 A KR 20257040002A KR 20250171479 A KR20250171479 A KR 20250171479A
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lys
glu
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asp
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Korean (ko)
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페러크 세이크
테츠야 카와무라
글로리아 모
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소흠, 인코포레이티드
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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    • 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)
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
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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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KR1020257040002A 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템 Pending KR20250171479A (ko)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201562140454P 2015-03-31 2015-03-31
US62/140,454 2015-03-31
US201562210451P 2015-08-27 2015-08-27
US62/210,451 2015-08-27
US201562240359P 2015-10-12 2015-10-12
US62/240,359 2015-10-12
KR1020247041475A KR102894634B1 (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템
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

Related Parent Applications (1)

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KR1020247041475A Division KR102894634B1 (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템

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KR20250171479A true KR20250171479A (ko) 2025-12-08

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KR1020257040002A Pending KR20250171479A (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템
KR1020177031337A Active KR102769515B1 (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템
KR1020247041475A Active KR102894634B1 (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템

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KR1020177031337A Active KR102769515B1 (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템
KR1020247041475A Active KR102894634B1 (ko) 2015-03-31 2016-03-31 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템

Country Status (8)

Country Link
US (2) US20180080051A1 (https=)
EP (2) EP3277805B1 (https=)
JP (4) JP2018513681A (https=)
KR (3) KR20250171479A (https=)
CN (1) CN108124453B (https=)
DK (1) DK3277805T3 (https=)
ES (1) ES3030433T3 (https=)
WO (1) WO2016161207A1 (https=)

Families Citing this family (76)

* Cited by examiner, † Cited by third party
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
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
JP2018513681A (ja) * 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
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
GB201610041D0 (en) * 2016-06-08 2016-07-20 Oxford Genetics Ltd Methods
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
US12188059B2 (en) 2016-09-13 2025-01-07 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
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
KR20190082318A (ko) * 2016-11-22 2019-07-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Crispr/cpf1 시스템 및 방법
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
JP7275043B2 (ja) 2016-12-16 2023-05-17 ビー－モーゲン・バイオテクノロジーズ，インコーポレーテッド 増大したｈＡＴファミリートランスポゾン媒介遺伝子導入ならびに関連する組成物、システムおよび方法
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
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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
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.
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
SG11201913852RA (en) 2017-07-07 2020-01-30 Toolgen Inc Target-specific crispr mutant
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
WO2019090175A1 (en) * 2017-11-02 2019-05-09 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
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CA3104288A1 (en) 2018-06-21 2019-12-26 B-Mogen Biotechnologies, Inc. Enhanced hat family transposon-mediated gene transfer and associated compositions, systems, and methods
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
AU2019365100B2 (en) * 2018-10-22 2025-10-09 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
CN113544266A (zh) * 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
JP7624728B2 (ja) * 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Ｄｎａ結合ドメイントランスアクチベーター及びその使用
BR112021017655A2 (pt) * 2019-03-07 2021-11-16 Univ Columbia Método e sistema de integração de dna guiada por rna usando transposons tipo tn7
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
KR20220030945A (ko) * 2019-05-23 2022-03-11 크리스티아나 케어 헬스 서비시즈 인코포레이티드 암의 치료를 위한 nrf2의 유전자 녹아웃
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
US20220235379A1 (en) * 2019-06-11 2022-07-28 Universitat Pompeu Fabra Targeted gene editing constructs and methods of using the same
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021097118A1 (en) * 2019-11-12 2021-05-20 The Broad Institute, Inc. Small type ii cas proteins and methods of use thereof
CN115867650A (zh) * 2020-04-20 2023-03-28 克里斯蒂安娜基因编辑研究公司 用于肺癌治疗的aav递送系统
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
KR102679172B1 (ko) * 2020-07-06 2024-06-28 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
WO2022020706A1 (en) * 2020-07-24 2022-01-27 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
JP2023553701A (ja) * 2020-12-16 2023-12-25 ウニベルシタット ポンペウ ファブラ 先天性筋ジストロフィーの処置のための治療用ｌａｍａ２ペイロード
AU2021403660A1 (en) * 2020-12-16 2023-07-06 Universitat Pompeu Fabra Programmable transposases and uses thereof
CN121712899A (zh) * 2023-04-27 2026-03-20 伦斯勒理工学院 用于在真核细胞中准确插入dna序列的重组酶
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 (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0871711A1 (en) * 1995-12-01 1998-10-21 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
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
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
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
ES2586210T3 (es) 2006-12-14 2016-10-13 Sangamo Biosciences, Inc. Proteínas de dedo de zinc no canónicas optimizadas
US8816153B2 (en) 2010-08-27 2014-08-26 Monsanto Technology Llc Recombinant DNA constructs employing site-specific recombination
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
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
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
EP2994531B1 (en) 2013-05-10 2018-03-28 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
WO2014191128A1 (en) * 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
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
JP2018513681A (ja) * 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

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