JP2024533311A - ゲノムを調節するための方法及び組成物 - Google Patents

ゲノムを調節するための方法及び組成物 Download PDF

Info

Publication number
JP2024533311A
JP2024533311A JP2024515067A JP2024515067A JP2024533311A JP 2024533311 A JP2024533311 A JP 2024533311A JP 2024515067 A JP2024515067 A JP 2024515067A JP 2024515067 A JP2024515067 A JP 2024515067A JP 2024533311 A JP2024533311 A JP 2024533311A
Authority
JP
Japan
Prior art keywords
domain
sequence
cas
identity
recombinant polypeptide
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
JP2024515067A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024533311A5 (https=
Inventor
ロバート チャールズ アルトシュラー，
アン ヘレン ボスマー，
セシリア ジョバンナ シルビア コッタ－ラムシノ，
ランディ ミシェル コトラー，
アナーニャ レイ，
ナサニエル ロケ，
カルロス サンチェス，
バレット イーサン スタインバーグ，
Original Assignee
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
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 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 filed Critical 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
Publication of JP2024533311A publication Critical patent/JP2024533311A/ja
Publication of JP2024533311A5 publication Critical patent/JP2024533311A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • 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
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024515067A 2021-09-08 2022-09-07 ゲノムを調節するための方法及び組成物 Pending JP2024533311A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163241953P 2021-09-08 2021-09-08
US63/241,953 2021-09-08
US202263373444P 2022-08-24 2022-08-24
US63/373,444 2022-08-24
PCT/US2022/076045 WO2023039424A2 (en) 2021-09-08 2022-09-07 Methods and compositions for modulating a genome

Publications (2)

Publication Number Publication Date
JP2024533311A true JP2024533311A (ja) 2024-09-12
JP2024533311A5 JP2024533311A5 (https=) 2025-09-16

Family

ID=85507644

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024515067A Pending JP2024533311A (ja) 2021-09-08 2022-09-07 ゲノムを調節するための方法及び組成物

Country Status (10)

Country Link
US (5) US12024728B2 (https=)
EP (1) EP4399292A4 (https=)
JP (1) JP2024533311A (https=)
KR (1) KR20260037664A (https=)
AU (1) AU2022343268A1 (https=)
CA (1) CA3231677A1 (https=)
IL (1) IL311225A (https=)
MX (1) MX2024002927A (https=)
TW (1) TW202323522A (https=)
WO (1) WO2023039424A2 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
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
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
WO2025024493A1 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
KR20260044217A (ko) 2023-07-25 2026-04-01 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 Cas 엔도뉴클레아제 및 관련 방법
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025090525A1 (en) * 2023-10-22 2025-05-01 Tessera Therapeutics, Inc. Lipid nanoparticles for delivery of therapeutic payloads to t cells
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025194138A1 (en) * 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2026015832A2 (en) * 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such

Family Cites Families (148)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US6699663B1 (en) 1995-12-14 2004-03-02 The General Hospital Corporation Molecular sequence of swine retrovirus
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
US6693086B1 (en) 1998-06-25 2004-02-17 National Jewish Medical And Research Center Systemic immune activation method using nucleic acid-lipid complexes
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
EP1083231A1 (en) 1999-09-09 2001-03-14 Introgene B.V. Smooth muscle cell promoter and uses thereof
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6689558B2 (en) 2000-02-08 2004-02-10 Sangamo Biosciences, Inc. Cells for drug discovery
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
US6627424B1 (en) 2000-05-26 2003-09-30 Mj Bioworks, Inc. Nucleic acid modifying enzymes
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US7169874B2 (en) 2001-11-02 2007-01-30 Bausch & Lomb Incorporated High refractive index polymeric siloxysilane compositions
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
JP4638225B2 (ja) 2002-05-30 2011-02-23 ザ スクリプス リサーチ インスティテュート 銅を触媒とするアジドとアセチレンのライゲーション
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
US9267932B2 (en) 2003-05-28 2016-02-23 The Johns Hopkins University Synthetic mammalian retrotransposon gene
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7070941B2 (en) 2003-11-17 2006-07-04 Board Of Regents, The University Of Texas System Methods and compositions for tagging via azido substrates
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
AU2005244258B2 (en) 2004-04-09 2010-07-01 Monsanto Technology Llc Compositions and methods for control of insect infestations in plants
DE102004034461B4 (de) 2004-07-16 2008-02-07 Chemotherapeutisches Forschungsinstitut Georg-Speyer-Haus Gentherapie solider Tumore durch retrovirale, mit Arenavirus-Glykoprotein pseudotypisierte Vektoren
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
US20110214199A1 (en) 2007-06-06 2011-09-01 Monsanto Technology Llc Genes and uses for plant enhancement
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
US8394604B2 (en) 2008-04-30 2013-03-12 Paul Xiang-Qin Liu Protein splicing using short terminal split inteins
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
MX360460B (es) 2008-10-20 2018-11-05 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
GB0901593D0 (en) 2009-01-30 2009-03-11 Touchlight Genetics Ltd Production of closed linear DNA
US10113156B2 (en) 2009-03-04 2018-10-30 Board Of Regents, The University Of Texas System Stabilized reverse transcriptase fusion proteins
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
AU2010325563B2 (en) 2009-11-27 2017-02-02 Basf Plant Science Company Gmbh Chimeric endonucleases and uses thereof
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
US8846883B2 (en) 2011-08-16 2014-09-30 University Of Southhampton Oligonucleotide ligation
EP3431497B1 (en) 2012-06-27 2022-07-27 The Trustees of Princeton University Split inteins, conjugates and uses thereof
CN102723640B (zh) 2012-07-09 2015-08-26 东莞中探探针有限公司 磁性连接器结构
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
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
EP3041940B1 (en) * 2013-09-02 2020-10-28 Cellectis Rna based method to obtain stably integrated retroviral vectors
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
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
JP2017503521A (ja) 2014-01-22 2017-02-02 ライフ テクノロジーズ コーポレーション 高温核酸合成で使用するための新規な逆転写酵素
US9879283B2 (en) 2014-10-09 2018-01-30 Life Technologies Corporation CRISPR oligonucleotides and gene editing
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
PT3408292T (pt) 2016-01-29 2023-07-19 Univ Princeton Inteínas divididas com excecional atividade de splicing
AU2017219605B2 (en) 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
WO2018045321A1 (en) 2016-09-02 2018-03-08 North Carolina State University Methods and compositions for modification of plastid genomes
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
US11280790B2 (en) 2016-10-04 2022-03-22 Svar Life Science Ab System and products for improved quantification of ADCC activity
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US20190330620A1 (en) 2016-10-14 2019-10-31 Emendobio Inc. Rna compositions for genome editing
US20180127759A1 (en) 2016-10-28 2018-05-10 Massachusetts Institute Of Technology Dynamic genome engineering
EP4545649A3 (en) 2016-11-11 2025-06-04 Bio-Rad Laboratories, Inc. Methods for processing nucleic acid samples
US9816093B1 (en) 2016-12-06 2017-11-14 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11667899B2 (en) 2017-06-27 2023-06-06 Yale University Reverse transcriptase and methods of use
CA3073448A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3679143A1 (en) 2017-09-08 2020-07-15 Life Technologies Corporation Methods for improved homologous recombination and compositions thereof
WO2019067910A1 (en) 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
WO2019070843A1 (en) 2017-10-03 2019-04-11 Board Of Regents, The University Of Texas System INVERSE TRANSCRIPTASE WITH LTR-FREE BACKLIGHTS AND USES THEREOF
CA3084185A1 (en) 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
JP2021506251A (ja) 2017-12-14 2021-02-22 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系、ならびにゲノム編集および他の適用におけるその使用
EP3728589A4 (en) 2017-12-22 2021-11-03 G+Flas Life Sciences CHEMICAL GENOMIC ENGINEERING MOLECULES AND PROCESSES
MA52116A (fr) 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
WO2019178428A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna and rna targeting enzymes and systems
EP3775239A4 (en) 2018-03-25 2022-01-05 Genetether, Inc MODIFIED NUCLEIC ACID EDITING SYSTEM FOR BINDING DONOR DNA
CN112533627A (zh) 2018-03-27 2021-03-19 G+Flas 生命科学公司 序列特异性体内细胞靶向
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
US20210355475A1 (en) 2018-08-10 2021-11-18 Cornell University Optimized base editors enable efficient editing in cells, organoids and mice
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
US20210079404A9 (en) 2018-09-25 2021-03-18 Massachusetts Institute Of Technology Engineered dcas9 with reduced toxicity and its use in genetic circuits
CN113710799B (zh) 2018-11-28 2024-11-12 克里斯珀医疗股份公司 用于在LNP中使用的编码CAS9的优化mRNA
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
US10947517B2 (en) * 2019-02-15 2021-03-16 Sigma-Aldrich Co. Llc CRISPR/Cas fusion proteins and systems
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020241869A1 (ja) * 2019-05-30 2020-12-03 国立大学法人東京大学 ２種の核酸塩基変換酵素が融合されたＣａｓタンパク質を利用したゲノム編集システム
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
US20220411777A1 (en) 2019-08-30 2022-12-29 The General Hospital Corporation C-to-G Transversion DNA Base Editors
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
CA3160186A1 (en) * 2019-11-05 2021-05-14 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
CA3162499A1 (en) 2019-11-22 2021-05-27 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
CN114829594B (zh) 2019-12-26 2025-03-21 新加坡科技研究局 核碱基编辑器
EP4085141A4 (en) * 2019-12-30 2024-03-06 The Broad Institute, Inc. GENOME EDITING USING REVERSE TRANSCRIPTASE FOR FULLY ACTIVE CRISPR COMPLEXES
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
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
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
CA3174553A1 (en) 2020-03-05 2021-09-10 Flagship Pioneering Innovations Vi, Llc Host defense suppressing methods and compositions for modulating a genome
JP2023518395A (ja) 2020-03-19 2023-05-01 インテリア セラピューティクス，インコーポレイテッド 指向性ゲノム編集のための方法及び組成物
EP4133069A2 (en) 2020-04-08 2023-02-15 Astrazeneca AB Compositions and methods for improved site-specific modification
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
AU2021403660A1 (en) 2020-12-16 2023-07-06 Universitat Pompeu Fabra Programmable transposases and uses thereof
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
CN116745428A (zh) 2021-01-14 2023-09-12 加利福尼亚大学董事会 位点特异性基因修饰
WO2022155532A1 (en) 2021-01-15 2022-07-21 4M Genomics Inc. Polypeptide fusions or conjugates for gene editing
WO2022158898A1 (ko) 2021-01-21 2022-07-28 한국생명공학연구원 Francisella novicida cas9 모듈 기반의 역전사 효소를 사용한 유전체 치환 및 삽입 기술
US20230374476A1 (en) 2021-02-05 2023-11-23 University Of Massachusetts Prime editor system for in vivo genome editing
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
EP4314257A4 (en) 2021-04-01 2025-07-09 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTIDE SEQUENCES
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
WO2023015014A1 (en) 2021-08-05 2023-02-09 Prime Medicine, Inc. Genome editing compositions and methods for treatment of myotonic dystrophy
WO2023015318A2 (en) 2021-08-05 2023-02-09 Prime Medicine, Inc. Genome editing compositions and methods for treatment of cystic fibrosis
JP2024530487A (ja) 2021-08-06 2024-08-21 ザ ブロード インスティテュート，インコーポレーテッド 改善されたプライムエディターおよび使用方法
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
MX2024002988A (es) 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
JP2024534945A (ja) 2021-09-10 2024-09-26 アジレント・テクノロジーズ・インク 化学修飾を有するプライム編集のためのガイドｒｎａ
EP4413128A1 (en) 2021-10-08 2024-08-14 The General Hospital Corporation Improved crispr prime editors
EP4419688A2 (en) 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
EP4430188A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of amyotrophic lateral sclerosis
EP4430185A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
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
CA3260587A1 (en) * 2022-06-24 2023-12-28 Flagship Pioneering Innovations Vi, Llc FAH COMPOSITIONS AND MODULATION METHODS

Also Published As

Publication number Publication date
US12123034B2 (en) 2024-10-22
US20240026324A1 (en) 2024-01-25
US20240002822A1 (en) 2024-01-04
KR20260037664A (ko) 2026-03-18
WO2023039424A3 (en) 2023-07-06
US20240247243A1 (en) 2024-07-25
US20240076638A1 (en) 2024-03-07
US20240002886A1 (en) 2024-01-04
EP4399292A2 (en) 2024-07-17
AU2022343268A1 (en) 2024-03-28
US12031162B2 (en) 2024-07-09
WO2023039424A2 (en) 2023-03-16
CA3231677A1 (en) 2023-03-16
MX2024002927A (es) 2024-05-29
IL311225A (en) 2024-05-01
TW202323522A (zh) 2023-06-16
US12037617B2 (en) 2024-07-16
US12024728B2 (en) 2024-07-02
EP4399292A4 (en) 2025-11-12

Similar Documents

Publication Publication Date Title
JP2024533311A (ja) ゲノムを調節するための方法及び組成物
JP2024533313A (ja) Ｈｂｂを調節する組成物及び方法
JP2024533312A (ja) Ｓｅｒｐｉｎａ調節組成物及び方法
JP2024533315A (ja) Ｐａｈ調節性組成物及び方法
EP4444362A2 (en) Cftr-modulating compositions and methods
JP2024533316A (ja) ゲノムを調節するための方法及び組成物
AU2023365692A1 (en) Improved gene editing systems utilizing trans recruiting components
JP2024533314A (ja) 遺伝子編集システム成分のｔｒａｎｓでの動員
JP2026509461A (ja) Ｓｅｒｐｉｎａ調節性組成物及び方法
WO2025235506A2 (en) Atp7b-modulating compositions and methods
EP4646479A2 (en) Trac and b2m modulating compositions and methods
CN118489007A (zh) 调节基因组的方法和组合物
WO2023225471A2 (en) Helitron compositions and methods
WO2025194124A1 (en) Modified st1cas9 guide nucleic acids
CN118556123A (zh) Hbb调节性组合物和方法
CN118613588A (zh) Serpina调节组合物和方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240514

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250905

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250905