AU2019336793B2 - RNA and DNA base editing via engineered ADAR recruitment - Google Patents

RNA and DNA base editing via engineered ADAR recruitment Download PDF

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AU2019336793B2
AU2019336793B2 AU2019336793A AU2019336793A AU2019336793B2 AU 2019336793 B2 AU2019336793 B2 AU 2019336793B2 AU 2019336793 A AU2019336793 A AU 2019336793A AU 2019336793 A AU2019336793 A AU 2019336793A AU 2019336793 B2 AU2019336793 B2 AU 2019336793B2
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rna
cases
domain
sequence
vector
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AU2019336793A1 (en
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Genghao CHEN
Kyle M. FORD
Dhruva KATREKAR
Prashant MALI
Dario Meluzzi
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University of California, San Diego
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University of California Berkeley
University of California, San Diego
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
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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
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    • C12N2750/14011—Parvoviridae
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  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
AU2019336793A 2018-09-06 2019-09-06 RNA and DNA base editing via engineered ADAR recruitment Active AU2019336793B2 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862728007P 2018-09-06 2018-09-06
US62/728,007 2018-09-06
US201862766433P 2018-10-17 2018-10-17
US62/766,433 2018-10-17
US201862773150P 2018-11-29 2018-11-29
US201862773146P 2018-11-29 2018-11-29
US62/773,146 2018-11-29
US62/773,150 2018-11-29
US201862780241P 2018-12-15 2018-12-15
US62/780,241 2018-12-15
PCT/US2019/050095 WO2020051555A1 (en) 2018-09-06 2019-09-06 Rna and dna base editing via engneered adar recruitment

Publications (2)

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AU2019336793A1 AU2019336793A1 (en) 2021-04-08
AU2019336793B2 true AU2019336793B2 (en) 2024-11-21

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US (2) US20220010333A1 (enExample)
EP (2) EP4332224A3 (enExample)
JP (1) JP7720623B6 (enExample)
CN (2) CN119193702A (enExample)
AU (1) AU2019336793B2 (enExample)
CA (1) CA3110998A1 (enExample)
GB (1) GB2590880A (enExample)
WO (1) WO2020051555A1 (enExample)

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WO2019104094A2 (en) 2017-11-21 2019-05-31 The Regents Of The University Of California Fusion proteins and methods for site-directed genome editing
CA3136735A1 (en) 2019-04-15 2020-10-22 Edigene Inc. Methods and compositions for editing rnas
AU2020313143B2 (en) * 2019-07-12 2024-04-04 Peking University Targeted RNA editing by leveraging endogenous adar using engineered RNAs
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
CA3159799A1 (en) * 2019-12-02 2021-06-10 Prashant MALI Engineering circular guide rnas
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IL293671A (en) * 2019-12-09 2022-08-01 Astellas Pharma Inc Antisense guide RNA with an additional functional region for target RNA editing
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JP2023529316A (ja) * 2020-05-26 2023-07-10 シェイプ セラピューティクス インコーポレイテッド ゲノム編集のための組成物及び方法
EP4158019A1 (en) * 2020-05-26 2023-04-05 Shape Therapeutics Inc. Methods and compositions relating to engineered guide systems for adenosine deaminase acting on rna editing
EP4158017A1 (en) * 2020-05-26 2023-04-05 Shape Therapeutics Inc. Engineered circular polynucleotides
KR20230035362A (ko) * 2020-07-06 2023-03-13 에디진 테라퓨틱스 (베이징) 인크. 개선된 rna 편집 방법
CN116209762A (zh) * 2020-07-30 2023-06-02 阿达尔克斯制药有限公司 Adar依赖性编辑组合物及其使用方法
CN115989041A (zh) * 2020-08-24 2023-04-18 波涛生命科学有限公司 经工程化为表达adar1的细胞和非人动物及其用途
WO2022078569A1 (en) * 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Artificial nucleic acids for rna editing
US20230399635A1 (en) * 2020-11-11 2023-12-14 Shape Therapeutics Inc. RNA-Editing Compositions and Methods of Use
CN116648508A (zh) * 2020-11-11 2023-08-25 塑造治疗公司 Rna编辑组合物和使用方法
EP4256050A4 (en) * 2020-12-01 2025-01-22 Shape Therapeutics Inc. High throughput rna-editing screening methods
US20240150754A1 (en) * 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
CA3220338A1 (en) * 2021-05-25 2022-12-01 Yiannis SAVVA Engineered guide rnas and polynucleotides
US20240279656A1 (en) * 2021-06-29 2024-08-22 Shape Therapeutics Inc. Engineered Guide RNAs and Polynucleotides
TW202321451A (zh) * 2021-08-18 2023-06-01 北京大學 工程化的 adar 招募 rnas 及其使用方法
JP2024533163A (ja) 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集
JP2023040852A (ja) * 2021-09-10 2023-03-23 隆光 矢野 －１フレームシフトを誘導するための１本鎖核酸分子及び組成物
US20230167441A1 (en) * 2021-11-30 2023-06-01 Minghong Zhong Segmented Nucleic Acids
US20250115908A1 (en) * 2022-02-07 2025-04-10 The Regents Of The University Of California Method of interfering with repetitive rna
US20250215426A1 (en) * 2022-04-05 2025-07-03 Astellas Gene Therapies, Inc. Compositions and methods for the treatment of muscular dystrophies
CN116732039B (zh) * 2023-08-03 2023-11-14 呈诺再生医学科技(北京)有限公司 一种促进rna序列在细胞内直接环化翻译的序列组合及其应用
CN117210435A (zh) * 2023-08-29 2023-12-12 苏州湃芮生物科技有限公司 一种用于调控rna甲基化修饰的编辑系统及其应用
CN121059628A (zh) * 2024-06-03 2025-12-05 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA
WO2026061377A1 (zh) * 2024-09-18 2026-03-26 时夕(广州)生物科技有限公司 Adar向导rna及其应用
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EP3847255A4 (en) 2024-01-10
EP4332224A2 (en) 2024-03-06
US20260092292A1 (en) 2026-04-02
CN112996912A (zh) 2021-06-18
JP7720623B6 (ja) 2025-09-18
EP3847255A1 (en) 2021-07-14
JP2021536244A (ja) 2021-12-27
GB2590880A (en) 2021-07-07
GB202104889D0 (en) 2021-05-19
WO2020051555A1 (en) 2020-03-12
JP7720623B2 (ja) 2025-08-08
AU2019336793A1 (en) 2021-04-08
CA3110998A1 (en) 2020-03-12
CN119193702A (zh) 2024-12-27
EP4332224A3 (en) 2024-06-05
US20220010333A1 (en) 2022-01-13

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