IL268763A - Targeted RNA mutations using repressor tRNAs and deaminases - Google Patents

Targeted RNA mutations using repressor tRNAs and deaminases

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Publication number
IL268763A
IL268763A IL26876319A IL26876319A IL268763A IL 268763 A IL268763 A IL 268763A IL 26876319 A IL26876319 A IL 26876319A IL 26876319 A IL26876319 A IL 26876319A IL 268763 A IL268763 A IL 268763A
Authority
IL
Israel
Prior art keywords
deaminases
rna targeting
suppressor trnas
mutations via
via suppressor
Prior art date
Application number
IL26876319A
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English (en)
Hebrew (he)
Original Assignee
Univ California
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 Univ California filed Critical Univ California
Publication of IL268763A publication Critical patent/IL268763A/en

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    • 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
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    • A61K38/53Ligases (6)
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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)
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    • 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
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
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    • 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

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • 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)
IL26876319A 2017-03-03 2019-08-18 Targeted RNA mutations using repressor tRNAs and deaminases IL268763A (en)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
IL268763A true IL268763A (en) 2019-10-31

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IL26876319A IL268763A (en) 2017-03-03 2019-08-18 Targeted RNA mutations using repressor tRNAs and deaminases

Country Status (7)

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

Families Citing this family (51)

* 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
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
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
BR112022017704A2 (pt) * 2020-03-04 2022-11-01 Suzhou Qi Biodesign Biotechnology Company Ltd Método e sistema de edição de genoma multiplex
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
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
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)

Family Cites Families (38)

* 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
US20170191057A1 (en) * 2014-02-05 2017-07-06 Regents Of The University Of California Rna editing biomarkers for diagnosis, pharmacological screening and prognostication in cancer
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
WO2016097212A1 (en) 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
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

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