JP2024534945A - 化学修飾を有するプライム編集のためのガイドｒｎａ - Google Patents

化学修飾を有するプライム編集のためのガイドｒｎａ Download PDF

Info

Publication number
JP2024534945A
JP2024534945A JP2024514728A JP2024514728A JP2024534945A JP 2024534945 A JP2024534945 A JP 2024534945A JP 2024514728 A JP2024514728 A JP 2024514728A JP 2024514728 A JP2024514728 A JP 2024514728A JP 2024534945 A JP2024534945 A JP 2024534945A
Authority
JP
Japan
Prior art keywords
pegrna
nucleotides
sequence
reverse transcriptase
editing
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
JP2024514728A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024534945A5 (https=
Inventor
ライアン，ダニエル・イー．
デリンジャー，ダグラス・ジェイ．
カイザー，ロバート
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.)
Agilent Technologies Inc
Original Assignee
Agilent Technologies 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 Agilent Technologies Inc filed Critical Agilent Technologies Inc
Publication of JP2024534945A publication Critical patent/JP2024534945A/ja
Publication of JP2024534945A5 publication Critical patent/JP2024534945A5/ja
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
    • C12N15/10—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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N2310/20—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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/316—Phosphonothioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024514728A 2021-09-10 2022-09-12 化学修飾を有するプライム編集のためのガイドｒｎａ Pending JP2024534945A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163243055P 2021-09-10 2021-09-10
US63/243,055 2021-09-10
PCT/US2022/076317 WO2023039586A1 (en) 2021-09-10 2022-09-12 Guide rnas with chemical modification for prime editing

Publications (2)

Publication Number Publication Date
JP2024534945A true JP2024534945A (ja) 2024-09-26
JP2024534945A5 JP2024534945A5 (https=) 2025-09-22

Family

ID=85507043

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024514728A Pending JP2024534945A (ja) 2021-09-10 2022-09-12 化学修飾を有するプライム編集のためのガイドｒｎａ

Country Status (8)

Country Link
US (2) US11884915B2 (https=)
EP (1) EP4399294A4 (https=)
JP (1) JP2024534945A (https=)
KR (1) KR20240055811A (https=)
CN (1) CN117957317A (https=)
AU (1) AU2022343300A1 (https=)
CA (1) CA3230927A1 (https=)
WO (1) WO2023039586A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
EP4402266A4 (en) * 2021-09-14 2025-11-05 Agilent Technologies Inc GUIDELINES FOR USING RNA WITH CHEMICAL MODIFICATIONS
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024259051A1 (en) * 2023-06-14 2024-12-19 The Children's Medical Center Corporation Systems and methods for modifying a polynucleotide
WO2025019675A1 (en) * 2023-07-18 2025-01-23 The Trustees Of Princeton University Prime editing system and uses thereof
WO2025038842A1 (en) * 2023-08-15 2025-02-20 The Children's Medical Center Corporation Systems and methods for modifying a polynucleotide
WO2025111452A2 (en) * 2023-11-21 2025-05-30 Prime Medicine, Inc. CHEMICAL MODIFICATIONS IN PEgRNA and ngRNAs
KR20250075782A (ko) * 2023-11-21 2025-05-29 경상국립대학교산학협력단 교정 정밀도가 향상된 프라임 에디팅 시스템 및 이의 용도
WO2025151814A1 (en) * 2024-01-10 2025-07-17 The Broad Institute, Inc. Use of prime editing in correcting mutations in cftr

Family Cites Families (169)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5032401A (en) 1989-06-15 1991-07-16 Alpha Beta Technology Glucan drug delivery system and adjuvant
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
US7371580B2 (en) 2001-08-24 2008-05-13 Agilent Technologies, Inc. Use of unstructured nucleic acids in assaying nucleic acid molecules
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
WO2005065419A2 (en) 2003-12-29 2005-07-21 Am Biosolutions Method of culturing cells
US7740861B2 (en) 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
DK2476689T3 (en) 2007-05-10 2016-01-18 Agilent Technologies Inc Thiocarbonbeskyttende groups for RNA synthesis
JP5558097B2 (ja) 2007-12-10 2014-07-23 国立大学法人京都大学 効率的な核初期化方法
CA2695590C (en) 2008-06-27 2018-01-09 Kyoto University Method of efficiently establishing induced pluripotent stem cells
US20100076183A1 (en) 2008-09-22 2010-03-25 Dellinger Douglas J Protected monomer and method of final deprotection for rna synthesis
CA2770412C (en) 2009-08-07 2018-09-11 Kyoto University Method of efficiently establishing induced pluripotent stem cells
US8871504B2 (en) 2009-09-02 2014-10-28 Kyoto University Method of selecting safe pluripotent stem cells
US9585920B2 (en) 2011-02-04 2017-03-07 Katherine Rose Kovarik Method and system for treating cancer cachexia
US9528124B2 (en) 2013-08-27 2016-12-27 Recombinetics, Inc. Efficient non-meiotic allele introgression
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
JP6351577B2 (ja) 2012-05-21 2018-07-04 アジレント・テクノロジーズ・インクＡｇｉｌｅｎｔ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． 組成物及びオリゴヌクレオチドをコンジュゲートする方法
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2014022702A2 (en) 2012-08-03 2014-02-06 The Regents Of The University Of California Methods and compositions for controlling gene expression by rna processing
US20140075593A1 (en) 2012-09-07 2014-03-13 Dow Agrosciences Llc Fluorescence activated cell sorting (facs) enrichment to generate plants
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
UA118090C2 (uk) 2012-09-07 2018-11-26 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив
US9914930B2 (en) 2012-09-07 2018-03-13 Dow Agrosciences Llc FAD3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
AU2013335451C1 (en) 2012-10-23 2024-07-04 Toolgen Incorporated Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof
KR102121086B1 (ko) 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
US20150315576A1 (en) 2012-11-01 2015-11-05 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
MX375143B (es) 2012-12-06 2025-03-06 Synthetic Genomics Inc Mutantes de alga que tienen un fenotipo aclimatado a alta luz bloqueado.
US9447422B2 (en) 2012-12-06 2016-09-20 Synthetic Genomics, Inc. Autonomous replication sequences and episomal DNA molecules
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
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
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093688A1 (en) 2012-12-12 2014-06-19 1Massachusetts Institute Of Technology Compositions and methods for functional nucleic acid delivery
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
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
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
WO2014159719A1 (en) 2013-03-14 2014-10-02 Scrips Health Methods of isolating nucleic acids
US20160138027A1 (en) 2013-03-14 2016-05-19 The Board Of Trustees Of The Leland Stanford Junior University Treatment of diseases and conditions associated with dysregulation of mammalian target of rapamycin complex 1 (mtorc1)
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
WO2014144155A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota Engineering plant genomes using crispr/cas systems
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
WO2014145736A2 (en) 2013-03-15 2014-09-18 Transposagen Biopharmaceuticals, Inc. Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm)
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
US20160040155A1 (en) 2013-04-16 2016-02-11 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
SG10201913068PA (en) 2013-06-04 2020-02-27 Harvard College Rna-guided transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
US9982277B2 (en) 2013-06-11 2018-05-29 The Regents Of The University Of California Methods and compositions for target DNA modification
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
ES2929143T3 (es) 2013-07-09 2022-11-25 Harvard College Ingeniería genómica guiada por ARN múltiplex
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
US20160143256A1 (en) 2013-07-10 2016-05-26 Joseph A. MAJZOUB Mrap2 knockouts
EP3666892A1 (en) 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
CN120574876A (zh) 2013-08-22 2025-09-02 纳幕尔杜邦公司 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法
US9765404B2 (en) 2013-09-04 2017-09-19 Dow Agrosciences Llc Rapid assay for identifying transformants having targeted donor insertion
EP3636750A1 (en) 2013-09-04 2020-04-15 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US20150064149A1 (en) 2013-09-04 2015-03-05 Mice With Horns, LLC. Materials and methods for correcting recessive mutations in animals
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150071946A1 (en) 2013-09-06 2015-03-12 The Johns Hopkins University Tumor-specific retrotransposon insertions
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015040075A1 (en) 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
US20150098954A1 (en) 2013-10-08 2015-04-09 Elwha Llc Compositions and Methods Related to CRISPR Targeting
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
AU2014331939A1 (en) 2013-10-08 2016-04-28 Amelia HENRY Drought-resistant cereal grasses and related materials and methods
UY35812A (es) 2013-11-04 2015-05-29 Dow Agrosciences Llc ?loci de maíz óptimos?.
JP6560203B2 (ja) 2013-11-04 2019-08-14 ダウ アグロサイエンシィズ エルエルシー 遺伝子ターゲッティングのためのユニバーサルドナー系
NZ746567A (en) 2013-11-04 2019-09-27 Dow Agrosciences Llc Optimal soybean loci
NZ719494A (en) 2013-11-04 2017-09-29 Dow Agrosciences Llc Optimal maize loci
CN105980395A (zh) 2013-11-04 2016-09-28 美国陶氏益农公司 最优大豆座位
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
US11326209B2 (en) 2013-11-07 2022-05-10 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
WO2015075056A1 (en) 2013-11-19 2015-05-28 Thermo Fisher Scientific Baltics Uab Programmable enzymes for isolation of specific dna fragments
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
WO2015089427A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
WO2015184259A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
US20150344836A1 (en) 2014-05-30 2015-12-03 Ohio State Innovation Foundation Agrobacterium Strains for Plant Transformation and Related Materials and Methods
ES2781323T3 (es) 2014-06-23 2020-09-01 Regeneron Pharma Montaje de ADN mediado por nucleasas
CN106604994B (zh) 2014-06-23 2021-12-14 通用医疗公司 通过测序评估的DSBs的全基因组无偏鉴定(GUIDE-Seq)
EP3164148A4 (en) 2014-06-25 2017-12-13 Cold Spring Harbor Laboratory Methods and compositions for inhibiting growth and epithelial to mesenchymal transition (emt) in cancer cells
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
EP3778891A1 (en) 2014-08-27 2021-02-17 New England Biolabs, Inc. Synthon formation
SG11201701245QA (en) 2014-08-27 2017-03-30 Caribou Biosciences Inc Methods for increasing cas9-mediated engineering efficiency
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
US11408007B2 (en) 2014-09-26 2022-08-09 Yale University Compositions and methods for biocontainment of microorganisms
CN107072165B (zh) 2014-09-26 2021-05-28 先锋国际良种公司 小麦Ms1多核苷酸、多肽以及使用方法
EP3198015A1 (en) 2014-09-26 2017-08-02 Philip Morris Products S.a.s. Reducing tobacco specific nitrosamines through alteration of the nitrate assimilation pathway
KR20170064540A (ko) 2014-09-26 2017-06-09 투 포어 가이즈, 인코포레이티드 합성 프로브의 나노포어 감지에 의한 표적 서열 검출
US9879283B2 (en) 2014-10-09 2018-01-30 Life Technologies Corporation CRISPR oligonucleotides and gene editing
JP6723230B2 (ja) 2014-10-09 2020-07-15 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 神経障害性疼痛を治療するためのｃｓｆ１−ｄａｐ１２経路のメンバー遺伝子の標的破壊
WO2016057755A1 (en) 2014-10-09 2016-04-14 Anthrogenesis Corporation Placenta-derived adherent cell exosomes and uses thereof
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
US20170335331A1 (en) 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
TWI716367B (zh) 2014-10-31 2021-01-21 麻省理工學院 用於常間回文重複序列叢集（ｃｒｉｓｐｒ）之大量平行組合性基因學
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
KR102888521B1 (ko) * 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
US9481696B1 (en) 2015-08-19 2016-11-01 Afton Chemical Corporation Thiophosphates and thiophosphate derivatives as lubricant additives
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
EP3390631B1 (en) 2015-12-18 2020-04-08 Danisco US Inc. Methods and compositions for t-rna based guide rna expression
US20190075770A1 (en) 2015-12-18 2019-03-14 Japan Science And Technology Agency Genetic modification non-human organism, egg cells, fertilized eggs, and method for modifying target genes
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
EP3390624A4 (en) 2015-12-18 2019-07-10 The Regents of The University of California Modified site-directed modifying polypeptides and methods of use thereof
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
WO2017106414A1 (en) 2015-12-18 2017-06-22 Danisco Us Inc. Methods and compositions for polymerase ii (pol-ii) based guide rna expression
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US20210277392A1 (en) 2017-11-02 2021-09-09 The Governors Of The University Of Alberta Chemically-modified guide rnas to improve crispr-cas protein specificity
WO2019126037A1 (en) 2017-12-19 2019-06-27 City Of Hope Modified tracrrnas grnas, and uses thereof
US20210363518A1 (en) 2018-03-19 2021-11-25 University Of Massachusetts Modified guide rnas for crispr genome editing
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2023283092A1 (en) * 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
WO2023288332A2 (en) * 2021-07-16 2023-01-19 Prime Medicine, Inc. Genome editing compositions and methods for treatment of wilson's disease
EP4373939A4 (en) * 2021-07-23 2025-11-19 Prime Medicine Inc Genome editing compositions and treatment methods for chronic granulomatous disease
EP4392039A4 (en) * 2021-08-24 2025-10-15 Prime Medicine Inc GENOME EDITING COMPOSITIONS AND METHODS FOR TREATING RETINOPATHY
CA3235827A1 (en) * 2021-10-21 2023-04-27 Wei Hsi Yeh Genome editing compositions and methods for treatment of retinitis pigmentosa
EP4419688A2 (en) * 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3

Also Published As

Publication number Publication date
CA3230927A1 (en) 2023-03-16
US20230092393A1 (en) 2023-03-23
CN117957317A (zh) 2024-04-30
US11884915B2 (en) 2024-01-30
EP4399294A4 (en) 2025-07-09
WO2023039586A1 (en) 2023-03-16
US20240218354A1 (en) 2024-07-04
AU2022343300A1 (en) 2024-04-18
EP4399294A1 (en) 2024-07-17
KR20240055811A (ko) 2024-04-29

Similar Documents

Publication Publication Date Title
AU2022204254B2 (en) Chemically modified guide rnas for crispr/cas-mediated gene regulation
US11884915B2 (en) Guide RNAs with chemical modification for prime editing
AU2022201266B2 (en) Targeted rna editing
US20230026726A1 (en) Crispr/cas-related methods and compositions for treating sickle cell disease
EP3129485A2 (en) Crispr/cas-related methods and compositions for treating cystic fibrosis
CA2981508A1 (en) Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US20230340468A1 (en) Methods for using guide rnas with chemical modifications
CA3230928A1 (en) Methods for using guide rnas with chemical modifications
CN118043465A (zh) 用于使用具有化学修饰的指导rna的方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250911

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250911