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 Los Angeles UCLA
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University of California San Diego UCSD
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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
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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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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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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    • C12N2310/50—Physical structure
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  • 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

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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) CN118416088A (enExample)
GB (1) GB2574769A (enExample)
IL (1) IL268763A (enExample)
WO (1) WO2018161032A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
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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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US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
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. SYSTEMS, PROCEDURES AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITING
WO2019090169A1 (en) 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna
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WO2019126709A1 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
JP7347830B2 (ja) * 2018-06-29 2023-09-20 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
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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
US10905778B2 (en) 2018-09-26 2021-02-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
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JP2022516919A (ja) * 2019-01-04 2022-03-03 ザ・ユニバーシティ・オブ・シカゴ Ｒｎａをモジュレートするためのシステムおよび方法
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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
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