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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Japanese (ja)
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JP2020508685A5 (enExample
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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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Publication of JP2020508685A publication Critical patent/JP2020508685A/ja
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Priority to JP2023023308A priority Critical patent/JP2023075118A/ja
Priority to JP2025008232A priority patent/JP2025066771A/ja
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    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • C12N15/115—Aptamers, 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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    • C12Y603/05—Carbon-nitrogen ligases with glutamine as amido-N-donor (6.3.5)
    • C12Y603/05007—Glutaminyl-tRNA synthase (glutamine-hydrolyzing) (6.3.5.7)
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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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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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

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JP2020508685A true JP2020508685A (ja) 2020-03-26
JP2020508685A5 JP2020508685A5 (enExample) 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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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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US (8) US11932856B2 (enExample)
EP (1) EP3589751A4 (enExample)
JP (3) JP2020508685A (enExample)
CN (2) CN110612353A (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 (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
JP6718872B2 (ja) * 2014-12-17 2020-07-08 プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップＰｒｏＱＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＩＩ Ｂ．Ｖ． 標的化ｒｎａ編集
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
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
WO2019084063A1 (en) * 2017-10-23 2019-05-02 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
AU2018360065A1 (en) 2017-11-02 2020-06-18 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with 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
CN112752844A (zh) * 2018-06-29 2021-05-04 蒂宾根大学 用于rna编辑的人工核酸
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020033601A1 (en) * 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel cas12b enzymes and systems
EP3844274A1 (en) 2018-08-28 2021-07-07 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
ES2962434T3 (es) * 2018-10-12 2024-03-19 Univ Beijing Procedimientos y composiciones para editar ARN
US12281338B2 (en) 2018-10-29 2025-04-22 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
EP3906042A4 (en) * 2019-01-04 2023-03-15 The University of Chicago RNA MODULATION SYSTEMS AND METHODS
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CA3130488A1 (en) 2019-03-19 2020-09-24 David R. Liu Methods and compositions for editing nucleotide sequences
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
EP3953469A1 (en) * 2019-04-11 2022-02-16 Arcturus Therapeutics, Inc. Synthetic transfer rna with extended anticodon loop
EP3956449A4 (en) 2019-04-15 2023-03-29 EdiGene Therapeutics (Beijing) Inc. METHODS AND COMPOSITIONS FOR EDITING RNAS
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3146771A1 (en) * 2019-07-12 2021-01-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20230089490A1 (en) * 2019-10-11 2023-03-23 University Of Massachusetts Raav-mediated in vivo delivery of suppressor trnas
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
WO2021113270A1 (en) 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
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
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
JP7755837B2 (ja) * 2020-03-12 2025-10-17 学校法人福岡大学 オリゴヌクレオチド及び標的ｒｎａの部位特異的編集方法
WO2021211762A2 (en) 2020-04-14 2021-10-21 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
EP4150076A1 (en) * 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4172336A4 (en) * 2020-06-29 2024-07-24 Bar Ilan University Composition and methods for identifying antisense guide rna for rna editing
WO2022056041A2 (en) * 2020-09-08 2022-03-17 The Regents Of The University Of California Rna and dna base editing via engineered adar
US20240150754A1 (en) 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
IL309025A (en) * 2021-06-15 2024-02-01 Massachusetts Inst Technology Deaminase-based RNA sensors
EP4396350A4 (en) 2021-09-03 2026-01-14 Tacit Therapeutics Inc RNA EDITING BY RECRUITMENT OF SPLICING COMPLEX COMPONENTS
US20250327092A1 (en) * 2022-03-30 2025-10-23 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells
WO2023205606A1 (en) 2022-04-18 2023-10-26 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
WO2024155741A1 (en) * 2023-01-18 2024-07-25 The Broad Institute, Inc. Prime editing-mediated readthrough of premature termination codons (pert)
WO2024155745A1 (en) * 2023-01-18 2024-07-25 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
WO2005094370A2 (en) 2004-03-29 2005-10-13 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
EP1784501B1 (en) 2004-05-14 2015-11-18 Rosetta Genomics Ltd VIRAL AND VIRUS ASSOCIATED MicroRNAS AND USES THEREOF
PL2049664T3 (pl) 2006-08-11 2012-02-29 Biomarin Tech Bv Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA
IL284530B (en) 2011-11-18 2022-07-01 Alnylam Pharmaceuticals Inc rnai factors, preparations and methods of using them for the treatment of transthyretin-related diseases
AU2013253074C1 (en) 2012-04-23 2018-06-14 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
ES2584856T3 (es) 2012-07-12 2016-09-29 Proqr Therapeutics Ii B.V. Oligonucleótidos para realizar un cambio en la secuencia de una molécula de arn diana presente en una célula viva
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
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
WO2016094845A2 (en) 2014-12-12 2016-06-16 Woolf Tod M Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
EP3288594B1 (en) * 2015-04-27 2022-06-29 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
CN114533900A (zh) 2015-10-09 2022-05-27 波涛生命科学有限公司 寡核苷酸组合物及其方法
WO2017201091A1 (en) 2016-05-16 2017-11-23 The Board Of Regents Of The University Of Texas System COMPOSITIONS FOR THE DELIVERY OF tRNA AS NANOPARTICLES AND METHODS OF USE THEREWITH
JP7074345B2 (ja) 2016-06-22 2022-05-24 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 一本鎖ｒｎａ編集オリゴヌクレオチド
IL264923B2 (en) 2016-09-01 2025-07-01 Proqr Therapeutics Ii Bv Single-stranded RNA-editing oligonucleotides with chemical modifications
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
GB2574769A (en) * 2017-03-03 2019-12-18 Univ California RNA Targeting of mutations via suppressor tRNAs and deaminases
KR102678809B1 (ko) 2017-05-18 2024-06-26 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법 및 조성물
WO2018237372A1 (en) 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
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
WO2019071048A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
CN118421665A (zh) 2017-10-06 2024-08-02 俄勒冈健康与科学大学 用于编辑rna的组合物和方法
US20210380955A1 (en) 2018-05-11 2021-12-09 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
CN112752844A (zh) 2018-06-29 2021-05-04 蒂宾根大学 用于rna编辑的人工核酸
JP2021531022A (ja) 2018-07-24 2021-11-18 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 環状ポリリボヌクレオチドを含む組成物及びその使用
US20220010333A1 (en) * 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
ES2962434T3 (es) 2018-10-12 2024-03-19 Univ Beijing Procedimientos y composiciones para editar ARN
CA3127243A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CA3126947A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
EP3914260A4 (en) 2019-01-22 2023-05-17 Korro Bio, Inc. RNA-EDITING OLIGONUCLEOTIDES AND THEIR USE

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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