JP2020508685A - サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング - Google Patents

サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング Download PDF

Info

Publication number
JP2020508685A
JP2020508685A JP2019547704A JP2019547704A JP2020508685A JP 2020508685 A JP2020508685 A JP 2020508685A JP 2019547704 A JP2019547704 A JP 2019547704A JP 2019547704 A JP2019547704 A JP 2019547704A JP 2020508685 A JP2020508685 A JP 2020508685A
Authority
JP
Japan
Prior art keywords
rna
adar
trna
protein
vector
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
JP2019547704A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020508685A5 (enExample
Inventor
マリ,プラシャント
カトレカール,ドゥルヴァ
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.)
University of California
University of California Berkeley
Original Assignee
University of California San Diego UCSD
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 University of California San Diego UCSD filed Critical University of California San Diego UCSD
Publication of JP2020508685A publication Critical patent/JP2020508685A/ja
Publication of JP2020508685A5 publication Critical patent/JP2020508685A5/ja
Priority to JP2023023308A priority Critical patent/JP2023075118A/ja
Priority to JP2025008232A priority patent/JP2025066771A/ja
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/53Ligases (6)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y603/00Ligases forming carbon-nitrogen bonds (6.3)
    • C12Y603/05Carbon-nitrogen ligases with glutamine as amido-N-donor (6.3.5)
    • C12Y603/05007Glutaminyl-tRNA synthase (glutamine-hydrolyzing) (6.3.5.7)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2795/00Bacteriophages
    • C12N2795/00011Details
    • C12N2795/18011Details ssRNA Bacteriophages positive-sense
    • C12N2795/18111Leviviridae
    • C12N2795/18122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2019547704A 2017-03-03 2018-03-02 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング Pending JP2020508685A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023023308A JP2023075118A (ja) 2017-03-03 2023-02-17 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
JP2025008232A JP2025066771A (ja) 2017-03-03 2025-01-21 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762466961P 2017-03-03 2017-03-03
US62/466,961 2017-03-03
US201762551732P 2017-08-29 2017-08-29
US62/551,732 2017-08-29
PCT/US2018/020762 WO2018161032A1 (en) 2017-03-03 2018-03-02 RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023023308A Division JP2023075118A (ja) 2017-03-03 2023-02-17 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング

Publications (2)

Publication Number Publication Date
JP2020508685A true JP2020508685A (ja) 2020-03-26
JP2020508685A5 JP2020508685A5 (enExample) 2021-07-26

Family

ID=63370240

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019547704A Pending JP2020508685A (ja) 2017-03-03 2018-03-02 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
JP2023023308A Pending JP2023075118A (ja) 2017-03-03 2023-02-17 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
JP2025008232A Pending JP2025066771A (ja) 2017-03-03 2025-01-21 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2023023308A Pending JP2023075118A (ja) 2017-03-03 2023-02-17 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
JP2025008232A Pending JP2025066771A (ja) 2017-03-03 2025-01-21 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング

Country Status (7)

Country Link
US (8) US11932856B2 (enExample)
EP (1) EP3589751A4 (enExample)
JP (3) JP2020508685A (enExample)
CN (2) CN118416088A (enExample)
GB (1) GB2574769A (enExample)
IL (1) IL268763A (enExample)
WO (1) WO2018161032A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023075118A (ja) * 2017-03-03 2023-05-30 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 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
JP2023552037A (ja) * 2020-10-12 2023-12-14 エバーハルト カール ウニヴェルジテート テュービンゲン Rna編集のための人工核酸
JP2024504608A (ja) * 2021-01-12 2024-02-01 北京大学 遺伝子操作rnaを使用した内因性adarの活用による標的化rnaの編集

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
PL3234134T3 (pl) * 2014-12-17 2020-12-28 Proqr Therapeutics Ii B.V. Ukierunkowana edycja rna
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3701042A4 (en) * 2017-10-23 2021-08-11 The Broad Institute, Inc. NUCLEIC ACID TARGETED EDITING SYSTEMS, METHODS AND COMPOSITIONS
MX2020004647A (es) 2017-11-02 2021-03-25 Wistar Inst Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna).
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US20230193242A1 (en) * 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US20220073915A1 (en) * 2018-06-29 2022-03-10 Eberhard-Karls-Universität-Tübingen Artificial nucleic acids for rna editing
WO2020014261A1 (en) 2018-07-09 2020-01-16 The Broad Institute, Inc. Rna programmable epigenetic rna modifiers and uses thereof
US20210163944A1 (en) * 2018-08-07 2021-06-03 The Broad Institute, Inc. Novel cas12b enzymes and systems
US20220160870A1 (en) 2018-08-28 2022-05-26 Roche Innovation Center Copenhagen A/S Neoantigen engineering using splice modulating compounds
US10905778B2 (en) 2018-09-26 2021-02-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
JP2022501055A (ja) * 2018-09-26 2022-01-06 ケース ウエスタン リザーブ ユニバーシティ 未成熟終止コドン媒介性障害を治療するための方法および組成物
WO2020074001A1 (en) * 2018-10-12 2020-04-16 Peking University Methods and Compositions for Editing RNAs
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020113135A1 (en) * 2018-11-29 2020-06-04 Flagship Pioneering Innovations V, Inc. Methods of modulating rna
AU2020204917A1 (en) * 2019-01-04 2021-08-19 The University Of Chicago Systems and methods for modulating RNA
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
BR112021018607A2 (pt) 2019-03-19 2021-11-23 Massachusetts Inst Technology Métodos e composições para editar sequências de nucleotídeos
CN111793627A (zh) * 2019-04-08 2020-10-20 中国科学院上海生命科学研究院 利用人工构建rna编辑酶进行rna定点编辑及相关应用
LU101182B1 (en) * 2019-04-11 2020-10-12 Univ Hamburg Synthetic transfer RNA with extended anticodon loop
JP2022528696A (ja) * 2019-04-11 2022-06-15 アークタラス テラピュウティクス、インク アンチコドンループを延長した合成転移rna
KR102851101B1 (ko) 2019-04-15 2025-08-27 에디진 테라퓨틱스(베이징) 인크. Rna를 편집하기 위한 방법 및 조성물
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
MX2022000497A (es) * 2019-07-12 2022-02-03 Univ Beijing Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente.
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021072201A1 (en) * 2019-10-11 2021-04-15 University Of Massachusetts Raav-mediated in vivo delivery of suppressor trnas
CA3163514A1 (en) * 2019-12-02 2021-06-10 David HUSS Targeted transfer rnas for treatment of diseases
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
CN111326211B (zh) * 2020-01-07 2023-12-19 深圳市早知道科技有限公司 一种检测地中海贫血基因变异的方法及检测装置
US20230053353A1 (en) * 2020-01-09 2023-02-23 The Regents Of The University Of California Targeting transfer rna for the suppression of nonsense mutations in messenger rna
US20240117368A1 (en) * 2020-03-04 2024-04-11 Suzhou Qi Biodesign Biotechnology Company Limited Multiplex genome editing method and system
WO2021182474A1 (ja) * 2020-03-12 2021-09-16 株式会社Frest オリゴヌクレオチド及び標的rnaの部位特異的編集方法
AU2021255583A1 (en) 2020-04-14 2022-11-10 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
WO2021231680A1 (en) * 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2022003684A1 (en) * 2020-06-29 2022-01-06 Bar-Ilan University Composition and methods for identifying antisense guide rna for rna editing
US20240150754A1 (en) 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
US12195734B2 (en) * 2021-06-15 2025-01-14 Massachusetts Institute Of Technology Deaminase-based RNA sensors
CA3230419A1 (en) 2021-09-03 2023-03-09 David A. Nelles Rna editing via recruitment of spliceosome components
EP4499848A2 (en) * 2022-03-30 2025-02-05 University of Pittsburgh - of the Commonwealth System of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells
AU2023255614A1 (en) 2022-04-18 2024-10-24 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver.
WO2023206088A1 (en) * 2022-04-26 2023-11-02 Huigene Therapeutics Co., Ltd. Rna base editor for treating dmd-associated diseases
CN114908066B (zh) * 2022-05-17 2024-01-23 杭州嵌化合生医药科技有限公司 一种正交翻译系统及其在再分配密码子恢复ptc疾病中功能蛋白表达方面的应用
EP4652272A1 (en) * 2023-01-18 2025-11-26 The Broad Institute Inc. Prime editing-mediated readthrough of premature termination codons (pert)
EP4652271A1 (en) * 2023-01-18 2025-11-26 The Broad Institute, Inc. Base editing-mediated readthrough of premature termination codons (bert)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002508959A (ja) * 1998-01-14 2002-03-26 ヒューマン ジーン セラピー リサーチ インスティテュート ヒトのサプレッサーtRNAオリゴヌクレオチド、及びそれを用いる方法
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
JP2016182140A (ja) * 2012-12-12 2016-10-20 ザ・ブロード・インスティテュート・インコーポレイテッ 配列操作のための改善された系、方法および酵素組成物のエンジニアリングおよび最適化
WO2017010556A1 (ja) * 2015-07-14 2017-01-19 学校法人福岡大学 部位特異的rna変異導入方法およびそれに使用する標的編集ガイドrnaならびに標的rna-標的編集ガイドrna複合体

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5759833A (en) * 1994-05-27 1998-06-02 Cubist Pharmaceuticals, Inc. Human isoleucyl-tRNA synthetase proteins, nucleic acids and tester strains comprising same
US8314226B2 (en) 2004-03-29 2012-11-20 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
IL179285A (en) 2004-05-14 2011-04-28 Rosetta Genomics Ltd Micrornas and uses thereof
DK2049664T3 (da) 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
US9399775B2 (en) 2011-11-18 2016-07-26 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
EP3228712A1 (en) 2012-04-23 2017-10-11 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
DK2852668T3 (en) 2012-07-12 2016-05-30 Proqr Therapeutics Ii Bv Oligonucleotides TO COMPLETE A CHANGE IN SEQUENCE OF A target RNA molecule THERE IS IN A LIVING CELL
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
RU2721275C2 (ru) * 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
WO2014204729A1 (en) * 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
WO2015120197A1 (en) 2014-02-05 2015-08-13 Regents Of The University Of California Rna editing biomarkers for diagnosis, pharmacological screening and prognostication in cancer
JP6997623B2 (ja) 2014-12-12 2022-02-04 エム. ウルフ、トッド オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法
WO2016176191A1 (en) * 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
DE102015012522B3 (de) 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
WO2017062862A2 (en) 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
PT3458074T (pt) 2016-05-16 2024-08-21 Univ Texas Composições para a entrega de trna na forma de nanopartículas e métodos de utilização com os mesmos
AU2017281497B2 (en) 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
PL3507366T3 (pl) 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
CN118416088A (zh) * 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CA3063739A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
EP3642342A4 (en) 2017-06-23 2021-03-17 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA AND TREATMENT METHODS
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
KR20250068785A (ko) 2017-10-04 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
US20200291383A1 (en) 2017-10-06 2020-09-17 Oregon Health & Science University Compositions and methods for editing rna
WO2019217943A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
US20220073915A1 (en) 2018-06-29 2022-03-10 Eberhard-Karls-Universität-Tübingen Artificial nucleic acids for rna editing
BR112021001343A2 (pt) 2018-07-24 2021-05-04 Flagship Pioneering Innovations Vi, Llc composições compreendendo poliribonucleotídeos circulares e seus usos
US20220010333A1 (en) * 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
WO2020074001A1 (en) 2018-10-12 2020-04-16 Peking University Methods and Compositions for Editing RNAs
CA3126947A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
JP2022518731A (ja) 2019-01-22 2022-03-16 コロ バイオ, インコーポレイテッド Rna編集オリゴヌクレオチド及びその使用
US11453878B2 (en) 2019-01-22 2022-09-27 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002508959A (ja) * 1998-01-14 2002-03-26 ヒューマン ジーン セラピー リサーチ インスティテュート ヒトのサプレッサーtRNAオリゴヌクレオチド、及びそれを用いる方法
JP2016182140A (ja) * 2012-12-12 2016-10-20 ザ・ブロード・インスティテュート・インコーポレイテッ 配列操作のための改善された系、方法および酵素組成物のエンジニアリングおよび最適化
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
WO2017010556A1 (ja) * 2015-07-14 2017-01-19 学校法人福岡大学 部位特異的rna変異導入方法およびそれに使用する標的編集ガイドrnaならびに標的rna-標的編集ガイドrna複合体

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ANGEW. CHEM. INT. ED. (2012), VOL.51, NO.44, PP.11166-11169, JPN6021050351, ISSN: 0004673000 *
SCIENCE (2016) VOL.351, NO.6271, PP.403-407, JPN6021050347, ISSN: 0005054176 *
SCIENTIFIC REPORTS (2017) VOL.7, NO.41478, PP.1-13, JPN6021050349, ISSN: 0004672999 *
生化学 (2016) VOL.88, NO.5, PP.555-556, JPN6021050353, ISSN: 0004673001 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023075118A (ja) * 2017-03-03 2023-05-30 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 サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
JP2023552037A (ja) * 2020-10-12 2023-12-14 エバーハルト カール ウニヴェルジテート テュービンゲン Rna編集のための人工核酸
JP2024504608A (ja) * 2021-01-12 2024-02-01 北京大学 遺伝子操作rnaを使用した内因性adarの活用による標的化rnaの編集

Also Published As

Publication number Publication date
US20200277607A1 (en) 2020-09-03
IL268763A (en) 2019-10-31
CN118416088A (zh) 2024-08-02
US20210198673A1 (en) 2021-07-01
JP2025066771A (ja) 2025-04-23
US20210163948A1 (en) 2021-06-03
US11608503B2 (en) 2023-03-21
CN110612353A (zh) 2019-12-24
US20240352465A1 (en) 2024-10-24
US11932856B2 (en) 2024-03-19
EP3589751A4 (en) 2021-11-17
GB201913819D0 (en) 2019-11-06
WO2018161032A1 (en) 2018-09-07
US20220186226A1 (en) 2022-06-16
JP2023075118A (ja) 2023-05-30
US20240392303A1 (en) 2024-11-28
US20200263180A1 (en) 2020-08-20
US20230139474A1 (en) 2023-05-04
US11479775B2 (en) 2022-10-25
GB2574769A (en) 2019-12-18
EP3589751A1 (en) 2020-01-08

Similar Documents

Publication Publication Date Title
JP2023075118A (ja) サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
US11702658B2 (en) Methods and compositions for editing RNAs
US20240417757A1 (en) Methods and compositions for modulating a genome
CN113631708B (zh) 编辑rna的方法和组合物
US20220010333A1 (en) Rna and dna base editing via engineered adar recruitment
US20200340012A1 (en) Crispr-cas genome engineering via a modular aav delivery system
JP2024511621A (ja) 新規crispr酵素、方法、システム、及びそれらの使用
US20240200104A1 (en) Ltr transposon compositions and methods
WO2022109275A2 (en) Vectors, systems and methods for eukaryotic gene editing
HK40081918A (en) Methods and compositions for editing rna
HK40081918B (en) Methods and compositions for editing rna
HK40056042B (en) Methods and compositions for editing rnas
HK40056042A (en) Methods and compositions for editing rnas
HK40061041A (en) Methods and compositions for editing rnas

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20191210

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20191210

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210302

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210302

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210527

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20211208

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220104

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220329

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220704

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20221018

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230217

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20230217

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20230228

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20230330

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20230404

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20230512

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250116