CN110612353A - 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 - Google Patents

经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 Download PDF

Info

Publication number
CN110612353A
CN110612353A CN201880029395.4A CN201880029395A CN110612353A CN 110612353 A CN110612353 A CN 110612353A CN 201880029395 A CN201880029395 A CN 201880029395A CN 110612353 A CN110612353 A CN 110612353A
Authority
CN
China
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
CN201880029395.4A
Other languages
English (en)
Chinese (zh)
Inventor
普拉尚特·马利
德拉瓦·卡特埃卡
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 San Diego UCSD
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
Priority to CN202410435171.0A priority Critical patent/CN118416088A/zh
Publication of CN110612353A publication Critical patent/CN110612353A/zh
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)
CN201880029395.4A 2017-03-03 2018-03-02 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 Pending CN110612353A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202410435171.0A CN118416088A (zh) 2017-03-03 2018-03-02 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762466961P 2017-03-03 2017-03-03
US62/466961 2017-03-03
US201762551732P 2017-08-29 2017-08-29
US62/551732 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
CN202410435171.0A Division CN118416088A (zh) 2017-03-03 2018-03-02 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向

Publications (1)

Publication Number Publication Date
CN110612353A true CN110612353A (zh) 2019-12-24

Family

ID=63370240

Family Applications (2)

Application Number Title Priority Date Filing Date
CN202410435171.0A Pending CN118416088A (zh) 2017-03-03 2018-03-02 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CN201880029395.4A Pending CN110612353A (zh) 2017-03-03 2018-03-02 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CN202410435171.0A Pending CN118416088A (zh) 2017-03-03 2018-03-02 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向

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
CN111326211A (zh) * 2020-01-07 2020-06-23 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
CN114908066A (zh) * 2022-05-17 2022-08-16 浙江大学 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
CN115667528A (zh) * 2020-03-04 2023-01-31 苏州齐禾生科生物科技有限公司 多重基因组编辑方法和系统

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
US11932856B2 (en) * 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
US20200332272A1 (en) * 2017-10-23 2020-10-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CN111712248B (zh) 2017-11-02 2024-07-26 衣阿华大学研究基金会 使用ACE-tRNA经由遗传重分配拯救终止密码子的方法
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3728575A4 (en) * 2017-12-22 2021-11-24 The Broad Institute, Inc. CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF DNA BASES
CN112752844A (zh) * 2018-06-29 2021-05-04 蒂宾根大学 用于rna编辑的人工核酸
WO2020033601A1 (en) * 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel cas12b enzymes and systems
WO2020043750A1 (en) 2018-08-28 2020-03-05 Roche Innovation Center Copenhagen A/S Neoantigen engineering using splice modulating compounds
AU2019350869A1 (en) * 2018-09-26 2021-05-20 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
US10905778B2 (en) 2018-09-26 2021-02-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
WO2020074001A1 (en) * 2018-10-12 2020-04-16 Peking University Methods and Compositions for Editing RNAs
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
EP3887516A1 (en) * 2018-11-29 2021-10-06 Flagship Pioneering Innovations V, Inc. Methods of modulating rna
US20220048962A1 (en) * 2019-01-04 2022-02-17 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
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
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
WO2020208169A1 (en) * 2019-04-11 2020-10-15 Universität Hamburg Synthetic transfer rna with extended anticodon loop
LU101182B1 (en) * 2019-04-11 2020-10-12 Univ Hamburg Synthetic transfer RNA with extended anticodon loop
CR20210572A (es) 2019-04-15 2022-04-07 Univ Beijing Métodos y composiciones para editar ácido ribonucleicos (arn)
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
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
CN115397983A (zh) * 2019-12-02 2022-11-25 西普治疗公司 用于治疗疾病的靶向转移rna
WO2021142343A1 (en) * 2020-01-09 2021-07-15 The Regents Of The University Of California Targeting transfer rna for the suppression of nonsense mutations in messenger rna
US20240247258A1 (en) * 2020-03-12 2024-07-25 Frest Inc. Oligonucleotide and target rna site-specific editing method
BR112022020780A2 (pt) 2020-04-14 2022-11-29 Flagship Pioneering Innovations Vi Llc Composições de trem e usos das mesmas
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
WO2022078569A1 (en) * 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Artificial nucleic acids for rna editing
US20240150754A1 (en) 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
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
JP2024523877A (ja) * 2021-06-15 2024-07-02 マサチューセッツ インスティテュート オブ テクノロジー デアミナーゼベースのrnaセンサー
MX2024002647A (es) 2021-09-03 2024-05-10 Tacit Therapeutics Inc Edición de arn a través del reclutamiento de componentes de espliceosomas.
IL316014A (en) * 2022-03-30 2024-11-01 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated virus vectors for delivering nucleic acids to retinal cells
TW202405173A (zh) 2022-04-18 2024-02-01 美商維泰克斯製藥公司 用於增強aav療法及降低aav向肝臟之趨性的組合物及方法
WO2023206088A1 (en) * 2022-04-26 2023-11-02 Huigene Therapeutics Co., Ltd. Rna base editor for treating dmd-associated diseases
EP4652271A1 (en) * 2023-01-18 2025-11-26 The Broad Institute, Inc. Base editing-mediated readthrough of premature termination codons (bert)
WO2024155741A1 (en) * 2023-01-18 2024-07-25 The Broad Institute, Inc. Prime editing-mediated readthrough of premature termination codons (pert)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing

Family Cites Families (36)

* 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
AU2225199A (en) * 1998-01-14 1999-08-02 Human Gene Therapy Research Institute Human suppressor trna oligonucleotides and methods of use for same
US8314226B2 (en) 2004-03-29 2012-11-20 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
CA2566519C (en) 2004-05-14 2020-04-21 Rosetta Genomics Ltd. Micrornas and uses thereof
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
PE20142362A1 (es) 2011-11-18 2015-01-30 Alnylam Pharmaceuticals Inc Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
HRP20171254T1 (hr) 2012-04-23 2018-01-12 Biomarin Technologies B.V. Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja
NZ703824A (en) 2012-07-12 2018-06-29 Proqr Therapeutics Ii Bv Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
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
EP4299741A3 (en) * 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
KR20160056869A (ko) * 2013-06-17 2016-05-20 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
CN107532162A (zh) 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
JP6851319B2 (ja) * 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系
US11390865B2 (en) 2015-07-14 2022-07-19 Fukuoka University Method for introducing site-directed RNA mutation, target editing guide RNA used in the method and target RNA-target editing guide RNA complex
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
JP7511326B2 (ja) 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
PL3458074T3 (pl) 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
NZ751483A (en) 2016-09-01 2022-07-01 Proqr Therapeutics Ii Bv Chemically modified single-stranded rna-editing oligonucleotides
WO2018134301A1 (en) 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
US11932856B2 (en) * 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
US11866697B2 (en) 2017-05-18 2024-01-09 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, CIRCULAR RNA PRODUCTION METHODS, AND PROCESSING METHODS
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
KR20250068785A (ko) 2017-10-04 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
EP3691747A4 (en) 2017-10-06 2021-10-06 Oregon Health & Science University RNA COMPOSITIONS AND EDITING PROCEDURES
CN119587720A (zh) 2018-05-11 2025-03-11 比姆医疗股份有限公司 利用可编程碱基编辑器系统编辑单核苷酸多态性的方法
CN112752844A (zh) 2018-06-29 2021-05-04 蒂宾根大学 用于rna编辑的人工核酸
AU2019312269A1 (en) 2018-07-24 2021-03-04 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
CA3110998A1 (en) * 2018-09-06 2020-03-12 The Regents Of The University Of California Rna and dna base editing via engneered adar recruitment
WO2020074001A1 (en) 2018-10-12 2020-04-16 Peking University Methods and Compositions for Editing RNAs
AU2020210645B2 (en) 2019-01-22 2025-04-24 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof
TW202043469A (zh) 2019-01-22 2020-12-01 美商科羅生物公司 Rna編輯之寡核苷酸及其用途
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
KUTTAN,A.等: "Mechanistic insights into editing-site specificity of ADARs", 《PNAS》 *
NELSON, C.E.等: "In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy", 《SCIENCE》 *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111326211A (zh) * 2020-01-07 2020-06-23 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
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
CN115667528A (zh) * 2020-03-04 2023-01-31 苏州齐禾生科生物科技有限公司 多重基因组编辑方法和系统
CN115667528B (zh) * 2020-03-04 2024-10-01 苏州齐禾生科生物科技有限公司 多重基因组编辑方法和系统
CN114908066A (zh) * 2022-05-17 2022-08-16 浙江大学 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
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

Also Published As

Publication number Publication date
US20210198673A1 (en) 2021-07-01
JP2025066771A (ja) 2025-04-23
EP3589751A4 (en) 2021-11-17
US20230139474A1 (en) 2023-05-04
CN118416088A (zh) 2024-08-02
JP2023075118A (ja) 2023-05-30
GB2574769A (en) 2019-12-18
US20240392303A1 (en) 2024-11-28
US11932856B2 (en) 2024-03-19
US11608503B2 (en) 2023-03-21
JP2020508685A (ja) 2020-03-26
US11479775B2 (en) 2022-10-25
US20210163948A1 (en) 2021-06-03
US20240352465A1 (en) 2024-10-24
EP3589751A1 (en) 2020-01-08
WO2018161032A1 (en) 2018-09-07
US20200277607A1 (en) 2020-09-03
US20200263180A1 (en) 2020-08-20
US20220186226A1 (en) 2022-06-16
IL268763A (en) 2019-10-31
GB201913819D0 (en) 2019-11-06

Similar Documents

Publication Publication Date Title
US20240352465A1 (en) RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
US12157898B2 (en) Methods and compositions for modulating a genome
CN113631708B (zh) 编辑rna的方法和组合物
US20230242899A1 (en) Methods and compositions for modulating a genome
KR20220004674A (ko) Rna를 편집하기 위한 방법 및 조성물
CN116209770A (zh) 用于调控基因组的改善的方法和组合物
US20200340012A1 (en) Crispr-cas genome engineering via a modular aav delivery system
CN119280261A (zh) 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法,包括遗传性疾病的治疗
JP2024504608A (ja) 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
US20240200104A1 (en) Ltr transposon compositions and methods
US20250333718A1 (en) Context-specific adenine base editors and uses thereof
JP2021522825A (ja) CRISPR/Cas9システムおよびその使用
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
CN116490610A (zh) 调控基因组的方法和组合物

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
RJ01 Rejection of invention patent application after publication

Application publication date: 20191224

RJ01 Rejection of invention patent application after publication