CA3174483A1 - Improved methods and compositions for modulating a genome - Google Patents

Improved methods and compositions for modulating a genome

Info

Publication number
CA3174483A1
CA3174483A1 CA3174483A CA3174483A CA3174483A1 CA 3174483 A1 CA3174483 A1 CA 3174483A1 CA 3174483 A CA3174483 A CA 3174483A CA 3174483 A CA3174483 A CA 3174483A CA 3174483 A1 CA3174483 A1 CA 3174483A1
Authority
CA
Canada
Prior art keywords
sequence
polypeptide
domain
dna
preceding embodiments
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3174483A
Other languages
English (en)
French (fr)
Inventor
Stephen Cleaver
Barrett Ethan Steinberg
Jacob Rosenblum RUBENS
Robert James Citorik
William Edward Salomon
Zi Jun WANG
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Flagship Pioneering Innovations VI Inc
Original Assignee
Flagship Pioneering Innovations VI Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Flagship Pioneering Innovations VI Inc filed Critical Flagship Pioneering Innovations VI Inc
Publication of CA3174483A1 publication Critical patent/CA3174483A1/en
Pending legal-status Critical Current

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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • 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]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • 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]
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Toys (AREA)
  • Cosmetics (AREA)
CA3174483A 2020-03-04 2021-03-04 Improved methods and compositions for modulating a genome Pending CA3174483A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3174483A1 true CA3174483A1 (en) 2021-09-10

Family

ID=77613826

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3174483A Pending CA3174483A1 (en) 2020-03-04 2021-03-04 Improved methods and compositions for modulating a genome

Country Status (10)

Country Link
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)
IL (1) IL296095A (enExample)
WO (1) WO2021178717A2 (enExample)

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

Also Published As

Publication number Publication date
EP4114941A4 (en) 2024-10-16
US12037602B2 (en) 2024-07-16
WO2021178717A9 (en) 2022-03-17
WO2021178717A2 (en) 2021-09-10
JP7863508B2 (ja) 2026-05-21
US20240076698A1 (en) 2024-03-07
AU2021230545A1 (en) 2022-09-29
US12065669B2 (en) 2024-08-20
JP2025172847A (ja) 2025-11-26
US20240018551A1 (en) 2024-01-18
US20240084333A1 (en) 2024-03-14
JP2023517294A (ja) 2023-04-25
US12565666B2 (en) 2026-03-03
IL296095A (en) 2022-11-01
BR112022017736A2 (pt) 2022-11-29
KR20230053735A (ko) 2023-04-21
CN116209770A (zh) 2023-06-02
EP4114941A2 (en) 2023-01-11

Similar Documents

Publication Publication Date Title
US20230076357A1 (en) Methods and Compositions for Directed Genome Editing
US12016908B2 (en) Compositions and methods for treating hemoglobinopathies
US20230332184A1 (en) Template guide rna molecules
CN114630904B (zh) 用于无毒调理的组合物和方法
CA3174486A1 (en) Methods and compositions for modulating a genome
EP4028026A1 (en) Novel nucleobase editors and methods of using same
CA3129988A1 (en) Methods and compositions for editing nucleotide sequences
CA3174537A1 (en) Methods and compositions for modulating a genome
CN114072496A (zh) 腺苷脱氨酶碱基编辑器及使用其修饰靶标序列中的核碱基的方法
CN120400115A (zh) 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法
US12129478B1 (en) Engineered adenosine deaminases and base editors thereof
CN120346348A (zh) 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法
AU2021230545A1 (en) Improved methods and compositions for modulating a genome
WO2021041885A2 (en) Compositions and methods for editing a mutation to permit transcription or expression
JP2025108504A (ja) プライマー認識が改良されたＰｈｉ２９ ＤＮＡポリメラーゼ変異体
CA3237317A1 (en) Direct replacement genome editing
US20240132868A1 (en) Compositions and methods for the self-inactivation of base editors
CN117561074A (zh) 腺苷脱氨酶变体及其用途
KR102876548B1 (ko) Francisella novicida Cas9 모듈 기반의 역전사 효소를 사용한 유전체 치환 및 삽입 기술
CA3133130A1 (en) High-precision base editors
EP4665406A1 (en) Crispr-transposon systems and components
CA3230629A1 (en) Viral guide rna delivery
CN120225674A (zh) 雷特综合征疗法

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD

Year of fee payment: 4

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250228

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20250303

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT

Effective date: 20250303

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250303

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250304

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250317

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250317

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250317

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20250429

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250512

R15 Change to inventor requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R15-R108 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRECT INVENTOR REQUEST RECEIVED

Effective date: 20251010

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251017

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260227

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260227

R16 Change to inventor recorded

Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R16-R110 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRECT INVENTOR REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20260415

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260415

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260415

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20260722