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 UCSD
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University of California Berkeley
University of California San Diego UCSD
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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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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  • 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)

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

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

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CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
WO2019104094A2 (en) 2017-11-21 2019-05-31 The Regents Of The University Of California Fusion proteins and methods for site-directed genome editing
EP3956449B1 (en) 2019-04-15 2026-02-25 EdiGene Therapeutics (Beijing) Inc. Methods and compositions for editing rnas
EP3997229A4 (en) * 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
WO2021113264A1 (en) * 2019-12-02 2021-06-10 The Regents Of The University Of California Engineering circular guide rnas
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
US20230059753A1 (en) * 2019-12-09 2023-02-23 Astellas Pharma Inc. Antisense guide rna with added functional region for editing target rna
CN115777020A (zh) * 2020-04-22 2023-03-10 塑造治疗公司 使用snrna组分的组合物和方法
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
JP2023528039A (ja) * 2020-05-26 2023-07-03 シェイプ セラピューティクス インコーポレイテッド 改変環状ポリヌクレオチド
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
US20230272379A1 (en) * 2020-07-06 2023-08-31 Edigene Therapeutics (Beijing ) Inc. Improved rna editing method
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
WO2022046667A1 (en) * 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
WO2022078569A1 (en) * 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Artificial nucleic acids for rna editing
JP2024500279A (ja) * 2020-11-11 2024-01-09 シェイプ セラピューティクス インコーポレイテッド Ｒｎａ編集組成物及び使用方法
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
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
EP4388089A4 (en) * 2021-08-18 2025-01-15 Peking University Engineered adar-recruiting rnas and methods of use thereof
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
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
WO2023196853A1 (en) * 2022-04-05 2023-10-12 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
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CA3110998A1 (en) 2020-03-12
JP7720623B2 (ja) 2025-08-08
EP4332224A2 (en) 2024-03-06
EP3847255A4 (en) 2024-01-10
EP3847255A1 (en) 2021-07-14
AU2019336793A1 (en) 2021-04-08
CN119193702A (zh) 2024-12-27
US20260092292A1 (en) 2026-04-02
GB202104889D0 (en) 2021-05-19
US20220010333A1 (en) 2022-01-13
EP4332224A3 (en) 2024-06-05
WO2020051555A1 (en) 2020-03-12
JP2021536244A (ja) 2021-12-27
GB2590880A (en) 2021-07-07
JP7720623B6 (ja) 2025-09-18
CN112996912A (zh) 2021-06-18

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