CA3230927A1 - Guide rnas with chemical modification for prime editing - Google Patents

Guide rnas with chemical modification for prime editing Download PDF

Info

Publication number
CA3230927A1
CA3230927A1 CA3230927A CA3230927A CA3230927A1 CA 3230927 A1 CA3230927 A1 CA 3230927A1 CA 3230927 A CA3230927 A CA 3230927A CA 3230927 A CA3230927 A CA 3230927A CA 3230927 A1 CA3230927 A1 CA 3230927A1
Authority
CA
Canada
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
CA3230927A
Other languages
English (en)
French (fr)
Inventor
Daniel E. Ryan
Douglas J. Dellinger
Robert Kaiser
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 CA3230927A1 publication Critical patent/CA3230927A1/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
    • C12N15/10—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)
CA3230927A 2021-09-10 2022-09-12 Guide rnas with chemical modification for prime editing Pending CA3230927A1 (en)

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 (1)

Publication Number Publication Date
CA3230927A1 true CA3230927A1 (en) 2023-03-16

Family

ID=85507043

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3230927A Pending CA3230927A1 (en) 2021-09-10 2022-09-12 Guide rnas with chemical modification for prime editing

Country Status (8)

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

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20260037664A (ko) 2021-09-08 2026-03-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하기 위한 방법 및 조성물
EP4402266A4 (en) * 2021-09-14 2025-11-05 Agilent Technologies Inc GUIDELINES FOR USING RNA WITH CHEMICAL MODIFICATIONS
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
WO2024259051A1 (en) * 2023-06-14 2024-12-19 The Children's Medical Center Corporation Systems and methods for modifying a polynucleotide
EP4747369A1 (en) * 2023-07-18 2026-05-27 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
KR102969401B1 (ko) * 2023-11-21 2026-05-29 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 (168)

* 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
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
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
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 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
PL2476689T3 (pl) 2007-05-10 2016-04-29 Agilent Technologies Inc Tiowęglowe grupy zabezpieczające do syntezy RNA
KR101532442B1 (ko) 2007-12-10 2015-06-29 고쿠리츠 다이가쿠 호진 교토 다이가쿠 효율적인 핵 초기화 방법
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
US8900871B2 (en) 2009-08-07 2014-12-02 Kyoto University Method of producing induced pluripotent stem cells using inhibitors of P53
JP5682043B2 (ja) 2009-09-02 2015-03-11 国立大学法人京都大学 安全な多能性幹細胞の選択方法
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
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
EP3747891A3 (en) 2012-05-21 2021-03-24 Agilent Technologies, Inc. Compositions and methods for conjugating oligonucleotides
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
HK1207111A1 (en) 2012-08-03 2016-01-22 加利福尼亚大学董事会 Methods and compositions for controlling gene expression by rna processing
BR112015004995B1 (pt) 2012-09-07 2023-05-02 Sangamo Biosciences, Inc. Método para modificação do genoma de uma célula, uso de uma célula, semente ou planta obtida pelo referido método e nuclease de dedo de zinco sítio específica
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
US20140075593A1 (en) 2012-09-07 2014-03-13 Dow Agrosciences Llc Fluorescence activated cell sorting (facs) enrichment to generate plants
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
EP4397759A3 (en) 2012-10-23 2024-10-09 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
US20150315576A1 (en) 2012-11-01 2015-11-05 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
CA2890110C (en) 2012-11-01 2023-05-02 Factor Bioscience Inc. Methods and products for expressing proteins in cells
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
CA2892821C (en) 2012-12-06 2022-04-26 Synthetic Genomics, Inc. Algal mutants having a locked-in high light acclimated phenotype
WO2014089513A1 (en) 2012-12-06 2014-06-12 Synthetic Genomics, Inc. Autonomous replication sequences and episomal dna molecules
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
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
WO2014093688A1 (en) 2012-12-12 2014-06-19 1Massachusetts Institute Of Technology Compositions and methods for functional nucleic acid delivery
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
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
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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst 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
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
ES2749624T3 (es) 2013-03-14 2020-03-23 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
WO2014159719A1 (en) 2013-03-14 2014-10-02 Scrips Health Methods of isolating nucleic acids
WO2014153118A1 (en) 2013-03-14 2014-09-25 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)
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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
JP2016512048A (ja) 2013-03-15 2016-04-25 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ ＣＲＩＳＰＲ／Ｃａｓシステムを使用した植物ゲノム操作
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
EP2971006A4 (en) 2013-03-15 2017-02-08 Transposagen Biopharmaceuticals, Inc. Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm)
ES2747833T3 (es) 2013-04-04 2020-03-11 Dartmouth College Composiciones y procedimientos para la escisión in vivo del ADN proviral del vih-1
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
EP2986709A4 (en) 2013-04-16 2017-03-15 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
EP2997146A4 (en) 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Methods and compositions for treatment of a genetic condition
JP6608807B2 (ja) 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
DK3004349T3 (en) 2013-05-29 2018-06-06 Cellectis Sa A method for producing precise DNA cleavage using CAS9 nickase activity
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
EP3603679B1 (en) 2013-06-04 2022-08-10 President and Fellows of Harvard College Rna-guided transcriptional regulation
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
EP3008181B1 (en) 2013-06-11 2019-11-06 The Regents of The University of California Methods and compositions for target dna modification
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
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
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
KR20260047646A (ko) 2013-07-09 2026-04-08 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 멀티플렉스 rna-가이드된 게놈 조작
WO2015006437A1 (en) 2013-07-10 2015-01-15 Majzoub Joseph A Mrap2 knockouts
CN105517579B (zh) 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
CN106133138A (zh) 2013-08-22 2016-11-16 先锋国际良种公司 使用引导多核苷酸/cas内切核酸酶系统的基因组修饰及其使用方法
KR20160048212A (ko) 2013-09-04 2016-05-03 씨에스아이알 유전자 발현을 침묵화시키기 위해 유전자 조절 인자들을 표적으로하는 부위-특이적인 뉴클레아제 단일 세포 분석법
WO2015035034A1 (en) 2013-09-04 2015-03-12 Mice With Horns, Llc Materials and methods for correcting recessive mutations in animals
BR102014021800B1 (pt) 2013-09-04 2022-03-29 Dow Agrosciences Llc Método para detectar a integração específica de sítio de uma sequência de doador de polinucleotídeo dentro de um sítio-alvo genômico
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150071946A1 (en) 2013-09-06 2015-03-12 The Johns Hopkins University Tumor-specific retrotransposon insertions
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
ES2681622T3 (es) 2013-09-18 2018-09-14 Kymab Limited Métodos, células y organismos
WO2015040075A1 (en) 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
US20150098954A1 (en) 2013-10-08 2015-04-09 Elwha Llc Compositions and Methods Related to CRISPR Targeting
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
US20160251675A1 (en) 2013-10-08 2016-09-01 International Rice Research Institute Drought-Resistant Cereal Grasses and Related Materials and Methods
CN105980395A (zh) 2013-11-04 2016-09-28 美国陶氏益农公司 最优大豆座位
WO2015066643A1 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal soybean loci
JP6633532B2 (ja) 2013-11-04 2020-01-22 ダウ アグロサイエンシィズ エルエルシー 最適なトウモロコシ遺伝子座
EP3066110B1 (en) 2013-11-04 2021-12-29 Corteva Agriscience LLC Optimal maize loci
BR102014027448A8 (pt) 2013-11-04 2021-08-24 Dow Agrosciences Llc cassete de doador de polinucleotídeo, uso de uma planta, de uma parte de planta, ou de uma célula vegetal compreendendo o referido cassete, e método para integração direcionada do mesmo
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
US11326209B2 (en) 2013-11-07 2022-05-10 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
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
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
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
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
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
CA2932439A1 (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
WO2015184262A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for 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
AU2015280069B2 (en) 2014-06-23 2021-08-12 The General Hospital Corporation Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq)
US9738897B2 (en) 2014-06-23 2017-08-22 Regeneron Pharmaceuticals, Inc. Nuclease-mediated DNA assembly
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
WO2015200725A1 (en) 2014-06-25 2015-12-30 Cold Spring Harbor Laboratory Methods and compositions for inhibiting growth and epithelial to mesenchymal transition (emt) in cancer cells
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP6594955B2 (ja) 2014-08-27 2019-10-23 ニユー・イングランド・バイオレイブス・インコーポレイテツド シントン形成
ES2730378T3 (es) 2014-08-27 2019-11-11 Caribou Biosciences Inc Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9
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
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
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
EP3197270B1 (en) 2014-09-26 2025-12-03 Pioneer Hi-Bred International, Inc. Wheat ms1 polynucleotides, polypeptides, and methods of use
WO2016073079A2 (en) 2014-09-26 2016-05-12 Yale University Compositions and methods for biocontainment of microorganisms
RU2721799C2 (ru) 2014-09-26 2020-05-22 Филип Моррис Продактс С.А. Уменьшение содержания табак-специфичных нитрозаминов посредством изменения пути ассимиляции нитратов
EP3198036A4 (en) 2014-09-26 2018-08-08 Two Pore Guys, Inc. Target sequence detection by nanopore sensing of synthetic probes
AU2015330855A1 (en) 2014-10-09 2017-04-27 Celularity Inc. Placenta-derived adherent cell exosomes and uses thereof
CA2963940A1 (en) 2014-10-09 2016-04-14 The Regents Of The University Of California Targeted disruption of a csf1-dap12 pathway member gene for the treatment of neuropathic pain
CN113930455A (zh) 2014-10-09 2022-01-14 生命技术公司 Crispr寡核苷酸和基因剪辑
KR102546296B1 (ko) 2014-10-31 2023-06-21 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 변형된 t 세포에서 유전자 발현의 변경 및 그의 용도
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
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
CN107787367B (zh) * 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导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
EP3392337B1 (en) 2015-12-18 2024-03-06 Japan Science and Technology Agency Genetic modification non-human organism, egg cells, fertilized eggs, and method for modifying target genes
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
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP3390632B1 (en) 2015-12-18 2025-09-10 Danisco US Inc. Methods and compositions for polymerase ii (pol-ii) based guide rna expression
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
EP3708665A1 (en) 2015-12-18 2020-09-16 Danisco US Inc. Methods and compositions for t-rna 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
US11939575B2 (en) 2017-12-19 2024-03-26 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
WO2020191241A1 (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
EP4370672A4 (en) * 2021-07-16 2025-07-09 Prime Medicine Inc GENOME EDITING COMPOSITIONS AND METHODS FOR TREATING WILSON'S DISEASE
WO2023004439A2 (en) * 2021-07-23 2023-01-26 Prime Medicine, Inc. Genome editing compositions and methods for treatment of 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
CA3235826A1 (en) * 2021-10-21 2023-04-27 Wei Hsi Yeh Genome editing compositions and methods for treatment of usher syndrome type 3

Also Published As

Publication number Publication date
US20230092393A1 (en) 2023-03-23
EP4399294A1 (en) 2024-07-17
JP2024534945A (ja) 2024-09-26
KR20240055811A (ko) 2024-04-29
EP4399294A4 (en) 2025-07-09
WO2023039586A1 (en) 2023-03-16
AU2022343300A1 (en) 2024-04-18
US11884915B2 (en) 2024-01-30
CN117957317A (zh) 2024-04-30
US20240218354A1 (en) 2024-07-04

Similar Documents

Publication Publication Date Title
US11884915B2 (en) Guide RNAs with chemical modification for prime editing
AU2022204254B2 (en) Chemically modified guide rnas for crispr/cas-mediated gene regulation
US20240425902A1 (en) Methods of purifying dna for gene synthesis
EP3122880B1 (en) Crispr/cas-related methods and compositions for treating sickle cell disease
US20230340468A1 (en) Methods for using guide rnas with chemical modifications
EP3129485A2 (en) Crispr/cas-related methods and compositions for treating cystic fibrosis
WO2022256578A2 (en) Circular guide rnas for crispr/cas editing systems
CA3079968A1 (en) Systems and methods for treating hyper-igm syndrome
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
JP2025526813A (ja) キャップ化ｍＲＮＡを調製する組成物及び方法
CN120290534A (zh) 腺嘌呤脱氨酶、包含其的碱基编辑器融合蛋白和碱基编辑器系统及其应用

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240918

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20240918

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240918

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250826

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250908

R00 Party data change recorded

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R00-R116 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPOINTMENT OF AGENT REQUEST

Effective date: 20250924

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R00-R119 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REVOCATION OF AGENT REQUEST

Effective date: 20250924

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: 20250924

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251027

R17 Change to representative recorded

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R17-R117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPOINTMENT OF AGENT REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20251208

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R17-R121 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REVOCATION OF AGENT REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20251208

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251208

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-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260721

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260721