KR20230053735A - 게놈의 조정을 위한 개선된 방법 및 조성물 - Google Patents

게놈의 조정을 위한 개선된 방법 및 조성물 Download PDF

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KR20230053735A
KR20230053735A KR1020227034440A KR20227034440A KR20230053735A KR 20230053735 A KR20230053735 A KR 20230053735A KR 1020227034440 A KR1020227034440 A KR 1020227034440A KR 20227034440 A KR20227034440 A KR 20227034440A KR 20230053735 A KR20230053735 A KR 20230053735A
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sequence
polypeptide
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스티븐 클리버
바레트 에단 스테인베르그
제이콥 로젠블룸 루벤스
로버트 제임스 시토리크
윌리엄 에드워드 살로몬
지 준 왕
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플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨
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KR1020227034440A 2020-03-04 2021-03-04 게놈의 조정을 위한 개선된 방법 및 조성물 Pending KR20230053735A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062985264P 2020-03-04 2020-03-04
US62/985,264 2020-03-04
US202063035674P 2020-06-05 2020-06-05
US63/035,674 2020-06-05
PCT/US2021/020943 WO2021178717A2 (en) 2020-03-04 2021-03-04 Improved methods and compositions for modulating a genome

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KR20230053735A true KR20230053735A (ko) 2023-04-21

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US (3) US12565666B2 (https=)
EP (1) EP4114941A4 (https=)
JP (2) JP2023517294A (https=)
KR (1) KR20230053735A (https=)
CN (1) CN116209770A (https=)
AU (1) AU2021230545A1 (https=)
BR (1) BR112022017736A2 (https=)
CA (1) CA3174483A1 (https=)
IL (1) IL296095A (https=)
WO (1) WO2021178717A2 (https=)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
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
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
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
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
CN114981409A (zh) 2019-09-03 2022-08-30 美洛德生物医药公司 用于基因组整合的方法和组合物
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
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
BR112023023642A2 (pt) 2021-05-11 2024-01-30 Myeloid Therapeutics Inc Métodos e composições para integração genômica
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
CN113755627B (zh) * 2021-09-24 2023-06-16 广西大学 一种基于顺式调控元件的引物及其设计方法和在分子标记中的应用
CN114277119B (zh) * 2021-12-09 2023-05-16 浙江大学医学院附属邵逸夫医院 一种环状RNA circ-Arsb在制备防治骨质疏松产品中的应用
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
WO2023212724A2 (en) * 2022-04-28 2023-11-02 Flagship Pioneering Innovations Vi, Llc Compositions and methods for modulating a genome in t cells, induced pluripotent stem cells, and respiratory epithelial cells
KR20250006975A (ko) * 2022-05-02 2025-01-13 더 리젠트스 오브 더 유니이버시티 오브 캘리포니아 부위 특이적 게놈 변형을 위한 다성분 시스템
CN116334141B (zh) * 2022-08-04 2026-04-24 北京中因科技股份有限公司 基于基因编辑的RHO-R135W-adRP基因编辑药物
CN116064797B (zh) * 2022-08-29 2023-10-20 广州达健生物科技有限公司 一种子宫内膜癌基因甲基化水平检测试剂及其应用
AU2023364078A1 (en) * 2022-10-19 2025-05-01 Metagenomi Therapeutics, Inc. Gene editing systems comprising reverse transcriptases
CN120418275A (zh) * 2022-12-19 2025-08-01 北京干细胞与再生医学研究院 一种在基因组插入大片段dna的系统
US12565665B2 (en) 2022-12-21 2026-03-03 Trustees Of Boston University Compositions and methods for controlled mRNA translation and stability
CN116179513B (zh) * 2023-03-10 2023-12-22 之江实验室 一种Cpf1蛋白及其在基因编辑中的应用
WO2024196963A2 (en) * 2023-03-21 2024-09-26 The Regents Of The University Of California Compositions and methods for genome editing
TW202449159A (zh) 2023-04-19 2024-12-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於產生文庫之組合物及方法
CN116523902B (zh) * 2023-06-21 2023-09-26 湖南盛鼎科技发展有限责任公司 基于改进yolov5的电子粉涂敷均匀检测方法及设备
WO2025006419A1 (en) 2023-06-26 2025-01-02 Flagship Pioneering Innovations Vii, Llc Engineered plasmodia and related methods
CN119286819A (zh) * 2023-07-10 2025-01-10 北京干细胞与再生医学研究院 工程化的哺乳动物基因写入系统

Family Cites Families (79)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030121063A1 (en) 1995-11-16 2003-06-26 The Trustees Of The University Of Pennsylvania Compositions and methods of use of mammalian retrotransposons
US6693086B1 (en) 1998-06-25 2004-02-17 National Jewish Medical And Research Center Systemic immune activation method using nucleic acid-lipid complexes
US20060183226A1 (en) 2002-01-31 2006-08-17 Haruhiko Fujiwara Methods for retrotransposing long interspersed elements (lines)
WO2004072290A1 (en) 2003-02-07 2004-08-26 The Administrators Of The Tulane Educational Fund Mammalian retrotransposable elements
JPWO2005054463A1 (ja) 2003-11-21 2007-12-06 財団法人大阪産業振興機構 レトロトランスポゾンを用いた哺乳動物のゲノム改変技術の開発
US20110171729A1 (en) 2006-05-04 2011-07-14 Ab Maxis Inc. Method for Producing Stable Mammalian Cell Lines Producing High Levels of Recombinant Proteins
EP2055784A1 (en) 2007-10-31 2009-05-06 Bundesrepublik Deutschland, letztvertreten durch den Präsidenten des Paul-Ehrlich-Instituts Prof. Dr. Johannes Löwer Controlled activation of non-LTR retrotransposons in mammals
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US20140113375A1 (en) 2012-10-21 2014-04-24 Lixin Liu Transient Expression And Reverse Transcription Aided Genome Alteration System
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3155116A4 (en) 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
EP3183358B1 (en) 2014-08-19 2020-10-07 President and Fellows of Harvard College Rna-guided systems for probing and mapping of nucleic acids
EP3188763B1 (en) 2014-09-02 2020-05-13 The Regents of The University of California Methods and compositions for rna-directed target dna modification
WO2016065364A1 (en) 2014-10-24 2016-04-28 Life Technologies Corporation Compositions and methods for enhancing homologous recombination
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
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
EP3356520B1 (en) 2015-10-02 2022-03-23 The U.S.A. as represented by the Secretary, Department of Health and Human Services Lentiviral protein delivery system for rna-guided genome editing
US11427837B2 (en) 2016-01-12 2022-08-30 The Regents Of The University Of California Compositions and methods for enhanced genome editing
US20170275665A1 (en) * 2016-02-24 2017-09-28 Board Of Regents, The University Of Texas System Direct crispr spacer acquisition from rna by a reverse-transcriptase-cas1 fusion protein
US11447768B2 (en) 2016-03-01 2022-09-20 University Of Florida Research Foundation, Incorporated Molecular cell diary system
WO2017173004A1 (en) 2016-03-30 2017-10-05 Mikuni Takayasu A method for in vivo precise genome editing
EP3443088B1 (en) 2016-04-13 2024-09-18 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
EP4166660A1 (en) 2016-04-29 2023-04-19 BASF Plant Science Company GmbH Improved methods for modification of target nucleic acids using fused guide rna - donor molecules
US20190345483A1 (en) 2016-05-12 2019-11-14 President And Fellows Of Harvard College AAV Split Cas9 Genome Editing and Transcriptional Regulation
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
WO2018045321A1 (en) 2016-09-02 2018-03-08 North Carolina State University Methods and compositions for modification of plastid genomes
ES2985812T3 (es) 2016-09-09 2024-11-07 The Board Of Trustees Of The Leland Stanfordjunior Univ Edición genómica de precisión de alto rendimiento
US20190330620A1 (en) 2016-10-14 2019-10-31 Emendobio Inc. Rna compositions for genome editing
EP4545649A3 (en) 2016-11-11 2025-06-04 Bio-Rad Laboratories, Inc. Methods for processing nucleic acid samples
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
EP3630849A4 (en) 2017-05-25 2021-01-13 The General Hospital Corporation BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
JP2021530212A (ja) 2018-07-13 2021-11-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California レトロトランスポゾンベースの送達媒体及びその使用方法
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
JP7658579B2 (ja) 2018-10-19 2025-04-08 ボード オブ リージェンツ，ザ ユニバーシティ オブ テキサス システム 操作された長鎖散在反復エレメント（ｌｉｎｅ）トランスポゾンおよびそれらの使用方法
WO2020186262A1 (en) 2019-03-14 2020-09-17 Cornell University Compositions and methods for gene targeting using crispr-cas and transposons
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220298495A1 (en) 2019-06-12 2022-09-22 Emendobio Inc. Novel genome editing tool
JP7759311B2 (ja) 2019-07-19 2025-10-23 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー リコンビナーゼ組成物及び使用方法
EP4034659A4 (en) 2019-09-27 2024-10-16 The Broad Institute, Inc. PROGRAMMABLE POLYNUCLEOTIDE EDITORS FOR AMPLIFIED HOMOLOGOUS RECOMBINATION
US20220411768A1 (en) 2019-10-21 2022-12-29 The Trustees Of Columbia University In The City Of New York Methods of performing rna templated genome editing
CA3162499A1 (en) 2019-11-22 2021-05-27 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
EP4085141A4 (en) 2019-12-30 2024-03-06 The Broad Institute, Inc. GENOME EDITING USING REVERSE TRANSCRIPTASE FOR FULLY ACTIVE CRISPR COMPLEXES
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
US20230348939A1 (en) 2020-03-04 2023-11-02 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
BR112022017713A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
CA3174553A1 (en) 2020-03-05 2021-09-10 Flagship Pioneering Innovations Vi, Llc Host defense suppressing methods and compositions for modulating a genome
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
WO2021248102A1 (en) 2020-06-05 2021-12-09 Flagship Pioneering Innovations Vi, Llc Template guide rna molecules
EP4274894A2 (en) 2021-01-11 2023-11-15 The Broad Institute, Inc. Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision
US20240132916A1 (en) 2021-02-09 2024-04-25 The Broad Institute, Inc. Nuclease-guided non-ltr retrotransposons and uses thereof
WO2022183210A1 (en) 2021-02-26 2022-09-01 Flagship Pioneering Innovations Vi, Llc Tissue-specific methods and compositions for modulating a genome
AU2022240747A1 (en) 2021-03-19 2023-10-05 Flagship Pioneering Innovations Vi, Llc Ltr transposon compositions and methods
EP4314257A4 (en) 2021-04-01 2025-07-09 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTIDE SEQUENCES
EP4347859A4 (en) 2021-05-26 2025-07-02 Flagship Pioneering Innovations Vi Llc INTEGRASE COMPOSITIONS AND METHODS
EP4352230A4 (en) 2021-06-03 2025-06-11 Prime Medicine, Inc. Genome editing compositions and methods for treating Wilson's disease
WO2023283092A1 (en) 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
EP4373939A4 (en) 2021-07-23 2025-11-19 Prime Medicine Inc Genome editing compositions and treatment methods for chronic granulomatous disease
WO2023015318A2 (en) 2021-08-05 2023-02-09 Prime Medicine, Inc. Genome editing compositions and methods for treatment of cystic fibrosis
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
IL311219A (en) 2021-09-08 2024-05-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for genome modulation
MX2024002988A (es) 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
CA3231679A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Hbb-modulating compositions and methods
EP4419688A2 (en) 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
EP4430185A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
EP4430188A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of amyotrophic lateral sclerosis
EP4430189A1 (en) 2021-11-11 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fragile x syndrome
IL313027A (en) 2021-11-24 2024-07-01 Prime Medicine Inc Modified prime editing guide rnas
EP4437106A2 (en) 2021-11-24 2024-10-02 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
EP4504921A4 (en) 2022-04-01 2026-04-15 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTID SEQUENCES

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