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

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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
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rna
adar
trna
protein
vector
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JP2020508685A5 (OSRAM
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マリ,プラシャント
カトレカール,ドゥルヴァ
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University of California
University of California Berkeley
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University of California San Diego UCSD
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Priority to JP2023023308A priority Critical patent/JP2023075118A/ja
Priority to JP2025008232A priority patent/JP2025066771A/ja
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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

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JP2020508685A5 JP2020508685A5 (OSRAM) 2021-07-26

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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

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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

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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)

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 (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
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
US20240150754A1 (en) 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
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)

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
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
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
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
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 (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

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