JP2025170240A5 - - Google Patents

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Publication number
JP2025170240A5
JP2025170240A5 JP2025122950A JP2025122950A JP2025170240A5 JP 2025170240 A5 JP2025170240 A5 JP 2025170240A5 JP 2025122950 A JP2025122950 A JP 2025122950A JP 2025122950 A JP2025122950 A JP 2025122950A JP 2025170240 A5 JP2025170240 A5 JP 2025170240A5
Authority
JP
Japan
Prior art keywords
adenosine deaminase
fusion protein
base editor
domain
seq
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
JP2025122950A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025170240A (ja
Filing date
Publication date
Priority claimed from PCT/US2020/018195 external-priority patent/WO2020168135A1/en
Priority claimed from JP2022514994A external-priority patent/JP7717684B2/ja
Application filed filed Critical
Publication of JP2025170240A publication Critical patent/JP2025170240A/ja
Publication of JP2025170240A5 publication Critical patent/JP2025170240A5/ja
Pending legal-status Critical Current

Links

JP2025122950A 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法 Pending JP2025170240A (ja)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201962897777P 2019-09-09 2019-09-09
US62/897,777 2019-09-09
USPCT/US2020/018195 2020-02-13
PCT/US2020/018195 WO2020168135A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating alpha-1 antitrypsin deficiency
JP2022514994A JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法
PCT/US2020/049975 WO2021050571A1 (en) 2019-09-09 2020-09-09 Novel nucleobase editors and methods of using same

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2022514994A Division JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法

Publications (2)

Publication Number Publication Date
JP2025170240A JP2025170240A (ja) 2025-11-18
JP2025170240A5 true JP2025170240A5 (enExample) 2026-03-09

Family

ID=74867196

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022514994A Active JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法
JP2025122950A Pending JP2025170240A (ja) 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2022514994A Active JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法

Country Status (8)

Country Link
US (1) US20230075877A1 (enExample)
EP (1) EP4028026A4 (enExample)
JP (2) JP7717684B2 (enExample)
KR (1) KR20220076467A (enExample)
CN (1) CN114667149A (enExample)
AU (1) AU2020344547A1 (enExample)
CA (1) CA3153624A1 (enExample)
WO (1) WO2021050571A1 (enExample)

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KR20250134703A (ko) 2018-05-11 2025-09-11 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
US12454694B2 (en) 2018-09-07 2025-10-28 Beam Therapeutics Inc. Compositions and methods for improving base editing
US12529041B2 (en) 2018-09-07 2026-01-20 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
WO2020168135A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
WO2022067089A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
EP4313118A4 (en) * 2021-03-26 2025-06-18 Beam Therapeutics Inc. ADENOSINE DEAMINASE VARIANTS AND THEIR USES
WO2022240858A1 (en) 2021-05-10 2022-11-17 Mammoth Biosciences, Inc. Effector proteins and methods of use
EP4363568A4 (en) * 2021-06-29 2025-12-03 Council Scient Ind Res MODIFIED FNCAS9 AND ITS USES
US20240352439A1 (en) * 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
JP2024533313A (ja) * 2021-09-08 2024-09-12 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 Hbbを調節する組成物及び方法
EP4409000A1 (en) 2021-09-28 2024-08-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
WO2023141590A2 (en) 2022-01-21 2023-07-27 Mammoth Biosciences, Inc. Effector proteins and methods of use
WO2023144104A1 (en) 2022-01-25 2023-08-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of βeta-thalassemia
AU2023248451A1 (en) 2022-04-04 2024-10-17 President And Fellows Of Harvard College Cas9 variants having non-canonical pam specificities and uses thereof
CN120456933A (zh) 2022-04-28 2025-08-08 布罗德研究所股份有限公司 编码碱基编辑器的aav载体及其用途
WO2023217888A1 (en) 2022-05-10 2023-11-16 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from βeta-thalassemia
JP2025517657A (ja) 2022-05-10 2025-06-10 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル シンシチン-1融合タンパク質及び標的細胞へのカーゴ送達のためのその使用
EP4558633A1 (en) 2022-07-22 2025-05-28 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from beta-thalassemia
CN115820691B (zh) * 2022-07-25 2023-08-22 安徽农业大学 一种基于LbCpf1变体的水稻碱基编辑系统和应用
CA3263821A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. ADVANCED CYTOSIN DESAMINASE AND DNA EDITING METHODS USING IT
CN115975986B (zh) * 2022-08-22 2023-08-08 山东舜丰生物科技有限公司 突变的Cas12j蛋白及其应用
CN119998447A (zh) * 2022-09-23 2025-05-13 基础科学研究院 新型腺嘌呤脱氨酶变体及使用其进行碱基编辑的方法
EP4662313A1 (en) 2023-02-06 2025-12-17 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through multiplex base editing
WO2024230760A1 (zh) * 2023-05-09 2024-11-14 北京齐禾生科生物科技有限公司 一种可作用于dna的腺苷脱氨酶及其应用
CN117965505A (zh) * 2023-06-28 2024-05-03 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器
AU2024299627A1 (en) 2023-07-25 2026-01-22 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025059520A1 (en) * 2023-09-15 2025-03-20 Beam Therapeutics Inc. Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
CN120536418A (zh) * 2024-02-26 2025-08-26 尧唐(上海)生物科技有限公司 一种脱氨酶变体、包含其的碱基编辑器及其应用

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CN108064282A (zh) * 2014-10-14 2018-05-22 哈洛齐梅公司 腺苷脱氨酶-2(ada2)、其变体的组合物及使用其的方法
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
US11242542B2 (en) * 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
AU2017342543B2 (en) * 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
EP3538561A4 (en) * 2016-11-11 2020-10-21 The Regents of The University of California RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE
KR20200121782A (ko) * 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
CN109957569B (zh) * 2017-12-22 2022-10-25 苏州齐禾生科生物科技有限公司 基于cpf1蛋白的碱基编辑系统和方法
CN109295186B (zh) * 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用

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