JP2025028857A5 - - Google Patents

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Publication number
JP2025028857A5
JP2025028857A5 JP2024192680A JP2024192680A JP2025028857A5 JP 2025028857 A5 JP2025028857 A5 JP 2025028857A5 JP 2024192680 A JP2024192680 A JP 2024192680A JP 2024192680 A JP2024192680 A JP 2024192680A JP 2025028857 A5 JP2025028857 A5 JP 2025028857A5
Authority
JP
Japan
Prior art keywords
cas9
terminal fragment
composition
terminal
deaminase
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024192680A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025028857A (ja
Filing date
Publication date
Priority claimed from PCT/US2019/050111 external-priority patent/WO2020051561A1/en
Application filed filed Critical
Publication of JP2025028857A publication Critical patent/JP2025028857A/ja
Publication of JP2025028857A5 publication Critical patent/JP2025028857A5/ja
Pending legal-status Critical Current

Links

JP2024192680A 2018-09-07 2024-11-01 核酸塩基編集システムを送達するための組成物および方法 Pending JP2025028857A (ja)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862728703P 2018-09-07 2018-09-07
US62/728,703 2018-09-07
US201862779404P 2018-12-13 2018-12-13
US62/779,404 2018-12-13
PCT/US2019/050111 WO2020051561A1 (en) 2018-09-07 2019-09-07 Compositions and methods for delivering a nucleobase editing system
JP2021512514A JP7657711B2 (ja) 2018-09-07 2019-09-07 核酸塩基編集システムを送達するための組成物および方法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021512514A Division JP7657711B2 (ja) 2018-09-07 2019-09-07 核酸塩基編集システムを送達するための組成物および方法

Publications (2)

Publication Number Publication Date
JP2025028857A JP2025028857A (ja) 2025-03-05
JP2025028857A5 true JP2025028857A5 (enExample) 2025-10-09

Family

ID=69722796

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021512514A Active JP7657711B2 (ja) 2018-09-07 2019-09-07 核酸塩基編集システムを送達するための組成物および方法
JP2024192680A Pending JP2025028857A (ja) 2018-09-07 2024-11-01 核酸塩基編集システムを送達するための組成物および方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2021512514A Active JP7657711B2 (ja) 2018-09-07 2019-09-07 核酸塩基編集システムを送達するための組成物および方法

Country Status (8)

Country Link
US (1) US20210301274A1 (enExample)
EP (1) EP3847254A4 (enExample)
JP (2) JP7657711B2 (enExample)
KR (1) KR20210055733A (enExample)
CN (1) CN112969790B (enExample)
AU (1) AU2019336245A1 (enExample)
CA (1) CA3112011A1 (enExample)
WO (1) WO2020051561A1 (enExample)

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Publication number Priority date Publication date Assignee Title
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
EP3799603A4 (en) 2018-05-11 2022-03-02 Beam Therapeutics, Inc. METHODS OF PATHOGENIC AMINO ACID SUBSTITUTION USING PROGRAMMABLE BASE EDITOR SYSTEMS
US20210301274A1 (en) * 2018-09-07 2021-09-30 Beam Therapeutics Inc. Compositions and Methods for Delivering a Nucleobase Editing System
US12454694B2 (en) 2018-09-07 2025-10-28 Beam Therapeutics Inc. Compositions and methods for improving base editing
JP2022536364A (ja) 2019-06-13 2022-08-15 ザ ジェネラル ホスピタル コーポレイション 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
WO2021188840A1 (en) * 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
GB2632564B (en) 2020-04-09 2025-06-18 Verve Therapeutics Inc Base editing of angptl3 and methods of using same for treatment of disease
WO2022020800A2 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
WO2022081890A1 (en) * 2020-10-14 2022-04-21 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
EP4377460A1 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
MX2024002927A (es) 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
EP4441074A2 (en) 2021-12-03 2024-10-09 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
WO2023169454A1 (zh) * 2022-03-08 2023-09-14 中国科学院遗传与发育生物学研究所 腺嘌呤脱氨酶及其在碱基编辑中的用途
WO2023225572A2 (en) 2022-05-17 2023-11-23 Nvelop Therapeutics, Inc. Compositions and methods for efficient in vivo delivery
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
CN120641436A (zh) * 2022-11-01 2025-09-12 纪念斯隆-凯特琳癌症中心 基于内含肽的分选系统和模块化嵌合多肽
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024233941A1 (en) * 2023-05-11 2024-11-14 Massachusetts Eye And Ear Infirmary Base editing approaches to treat abca4-associated stargardt disease
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025038955A1 (en) * 2023-08-16 2025-02-20 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Systems and methods for improving safety of split intein aav mediated gene therapy
CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025194124A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. Modified st1cas9 guide nucleic acids
WO2025243196A1 (en) * 2024-05-21 2025-11-27 Fondazione Istituto Italiano Di Tecnologia Gene regulation system (tuser)

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613854A1 (en) * 2014-03-05 2020-02-26 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
AU2015298571B2 (en) * 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016112242A1 (en) * 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
CN106011104B (zh) * 2015-05-21 2019-09-27 清华大学 利用拆分Cas系统进行基因编辑和表达调控方法
WO2016205759A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
JP2019524162A (ja) * 2016-08-18 2019-09-05 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 モジュラーAAV送達システムによるCRISPR−Casゲノム編集
GB2573062A (en) * 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
US20210301274A1 (en) * 2018-09-07 2021-09-30 Beam Therapeutics Inc. Compositions and Methods for Delivering a Nucleobase Editing System

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