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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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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KR1020227034440A 2020-03-04 2021-03-04 게놈의 조정을 위한 개선된 방법 및 조성물 Pending KR20230053735A (ko)

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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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US (3) US12565666B2 (enExample)
EP (1) EP4114941A4 (enExample)
JP (2) JP7863508B2 (enExample)
KR (1) KR20230053735A (enExample)
CN (1) CN116209770A (enExample)
AU (1) AU2021230545A1 (enExample)
BR (1) BR112022017736A2 (enExample)
CA (1) CA3174483A1 (enExample)
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Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2018165504A1 (en) 2017-03-09 2018-09-13 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
AU2019327449B2 (en) 2018-08-28 2026-04-09 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
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
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
WO2021046243A2 (en) 2019-09-03 2021-03-11 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
BR112022017715A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
IL296095A (en) 2020-03-04 2022-11-01 Flagship Pioneering Innovations Vi Llc Improved methods and compositions for genome modulation
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
WO2022241029A1 (en) 2021-05-11 2022-11-17 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
KR20260037664A (ko) 2021-09-08 2026-03-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하기 위한 방법 및 조성물
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
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
CA3256516A1 (en) * 2022-04-28 2023-11-02 Flagship Pioneering Innovations Vi, Llc Compositions and methods for modulating a genome in T lymphocytes, induced pluripotent stem cells, and respiratory epithelial cells
WO2023215727A2 (en) * 2022-05-02 2023-11-09 The Regents Of The University Of California Multicomponent systems for site-specific genome modifications
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
WO2024131786A1 (zh) * 2022-12-19 2024-06-27 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
WO2024220741A1 (en) 2023-04-19 2024-10-24 Flagship Pioneering Innovations Vii, Llc Compositions and methods for the production of libraries
CN121889499A (zh) * 2023-06-15 2026-04-17 比姆医疗股份有限公司 用于治疗α1-抗胰蛋白酶缺乏症的方法和组合物
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
CN121358858A (zh) * 2023-07-10 2026-01-16 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
CN117289601B (zh) * 2023-09-07 2026-08-04 杭州知衣科技有限公司 一种基于cnn-ga的自抗扰控制器参数优化方法

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)
US20070037759A1 (en) 2003-02-07 2007-02-15 Administrators Of The Tulane Educational Fund, The Mammalian retrotransposable elements
US20080104723A1 (en) 2003-11-21 2008-05-01 Osaka Industrial Promojtion Organization C/O Mydom Development of Mammalian Genome Modification Technique Using Retrotransposon
WO2007130543A2 (en) 2006-05-04 2007-11-15 Abmaxis 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
EP3064585B1 (en) 2012-12-12 2020-02-05 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
CN105209621B (zh) 2012-12-12 2021-05-25 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
ES2749624T3 (es) 2013-03-14 2020-03-23 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
CN111218447B (zh) 2013-11-07 2024-10-11 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
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
WO2016028843A2 (en) 2014-08-19 2016-02-25 President And Fellows Of Harvard College Rna-guided systems for probing and mapping of nucleic acids
WO2016036754A1 (en) 2014-09-02 2016-03-10 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
US11911415B2 (en) 2015-06-09 2024-02-27 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for improving transplantation
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2017059241A1 (en) 2015-10-02 2017-04-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Lentiviral protein delivery system for rna-guided genome editing
WO2017123609A1 (en) 2016-01-12 2017-07-20 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
WO2017180711A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
KR102370675B1 (ko) 2016-04-29 2022-03-04 바스프 플랜트 사이언스 컴퍼니 게엠베하 표적 핵산의 변형을 위한 개선된 방법
WO2017197238A1 (en) 2016-05-12 2017-11-16 President And Fellows Of Harvard College Aav split cas9 genome editing and transcriptional regulation
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
WO2018045321A1 (en) 2016-09-02 2018-03-08 North Carolina State University Methods and compositions for modification of plastid genomes
EP4431607A3 (en) 2016-09-09 2024-12-11 The Board of Trustees of the Leland Stanford Junior University High-throughput precision genome editing
US20190330620A1 (en) 2016-10-14 2019-10-31 Emendobio Inc. Rna compositions for genome editing
WO2018089860A1 (en) 2016-11-11 2018-05-17 2D Genomics Inc. Methods for processing nucleic acid samples
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
CA3063733A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
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
AU2019327449B2 (en) 2018-08-28 2026-04-09 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
CN117925571A (zh) 2018-10-19 2024-04-26 德克萨斯州大学系统董事会 工程化长散在元件(line)转座子及其使用方法
US12410429B2 (en) 2019-03-14 2025-09-09 Cornell University Compositions and methods for gene targeting using CRISPR-Cas and transposons
WO2020191241A1 (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
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
EP4034659A4 (en) 2019-09-27 2024-10-16 The Broad Institute, Inc. PROGRAMMABLE POLYNUCLEOTIDE EDITORS FOR AMPLIFIED HOMOLOGOUS RECOMBINATION
WO2021080922A1 (en) 2019-10-21 2021-04-29 The Trustees Of Columbia University In The City Of New York Methods of performing rna templated genome editing
EP4061940A4 (en) 2019-11-22 2024-10-23 Flagship Pioneering Innovations VI, LLC RECOMBINASE COMPOSITIONS AND METHODS OF USE
WO2021138469A1 (en) 2019-12-30 2021-07-08 The Broad Institute, Inc. Genome editing using reverse transcriptase enabled and fully active crispr complexes
IL296095A (en) 2020-03-04 2022-11-01 Flagship Pioneering Innovations Vi Llc Improved methods and compositions for genome modulation
US20230348939A1 (en) 2020-03-04 2023-11-02 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
BR112022017715A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
AU2021232005A1 (en) 2020-03-04 2022-09-29 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
BR112022017735A2 (pt) 2020-03-05 2022-11-29 Flagship Pioneering Innovations Vi Llc Métodos e composições de supressão de defesa de hospedeiro para modular um genoma
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
WO2021248102A1 (en) 2020-06-05 2021-12-09 Flagship Pioneering Innovations Vi, Llc Template guide rna molecules
AU2022206476A1 (en) 2021-01-11 2023-07-20 President And Fellows Of Harvard College 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
WO2023004439A2 (en) 2021-07-23 2023-01-26 Prime Medicine, Inc. Genome editing compositions and methods for treatment of chronic granulomatous disease
WO2023015318A2 (en) 2021-08-05 2023-02-09 Prime Medicine, Inc. Genome editing compositions and methods for treatment of cystic fibrosis
KR20260037664A (ko) 2021-09-08 2026-03-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하기 위한 방법 및 조성물
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
WO2023039447A2 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Serpina-modulating compositions and methods
MX2024002930A (es) 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
EP4399307A4 (en) 2021-09-08 2026-01-21 Flagship Pioneering Innovations Vi Llc HBB COMPOSITIONS AND MODULATION METHODS
CA3235826A1 (en) 2021-10-21 2023-04-27 Wei Hsi Yeh Genome editing compositions and methods for treatment of usher syndrome type 3
US20250319202A1 (en) 2021-11-09 2025-10-16 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
US20250011766A1 (en) 2021-11-09 2025-01-09 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
US20250297248A1 (en) 2021-11-24 2025-09-25 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
AU2022398241A1 (en) 2021-11-24 2024-06-06 Prime Medicine, Inc. Modified prime editing guide rnas
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
WO2023192655A2 (en) 2022-04-01 2023-10-05 Prime Medicine, Inc. Methods and compositions for editing nucleotide sequences

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US20240076698A1 (en) 2024-03-07
AU2021230545A1 (en) 2022-09-29
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US20240084333A1 (en) 2024-03-14
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CA3174483A1 (en) 2021-09-10
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