JP7679311B2 - プログラム可能塩基エディターシステムを用いた一塩基多型編集法 - Google Patents

プログラム可能塩基エディターシステムを用いた一塩基多型編集法 Download PDF

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JP7679311B2
JP7679311B2 JP2021568855A JP2021568855A JP7679311B2 JP 7679311 B2 JP7679311 B2 JP 7679311B2 JP 2021568855 A JP2021568855 A JP 2021568855A JP 2021568855 A JP2021568855 A JP 2021568855A JP 7679311 B2 JP7679311 B2 JP 7679311B2
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adenosine deaminase
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ジェイソン マイケル ゲールケ、
ナタリー ペトロシャン、
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ビーム セラピューティクス インク．
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JP2021568855A 2019-05-21 2020-05-20 プログラム可能塩基エディターシステムを用いた一塩基多型編集法 Active JP7679311B2 (ja)

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Application Number Priority Date Filing Date Title
US201962850919P 2019-05-21 2019-05-21
US62/850,919 2019-05-21
PCT/US2020/033807 WO2020236936A1 (en) 2019-05-21 2020-05-20 Methods of editing a single nucleotide polymorphism using programmable base editor systems

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JP2022533673A JP2022533673A (ja) 2022-07-25
JP2022533673A5 JP2022533673A5 (enExample) 2023-05-29
JP7679311B2 true JP7679311B2 (ja) 2025-05-19

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US (1) US20220387622A1 (enExample)
EP (1) EP3972654A4 (enExample)
JP (1) JP7679311B2 (enExample)
KR (1) KR20220010540A (enExample)
CN (2) CN114206395B (enExample)
AU (1) AU2020279751A1 (enExample)
CA (1) CA3140093A1 (enExample)
WO (1) WO2020236936A1 (enExample)

Families Citing this family (18)

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Publication number Priority date Publication date Assignee Title
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3924479A4 (en) * 2019-02-13 2023-01-25 Beam Therapeutics, Inc. ADENOSINE DEAMINASE BASE EDITORS AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE IN A TARGET SEQUENCE
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
EP4396207A4 (en) * 2021-09-03 2025-07-16 Univ Chicago POLYPEPTIDES AND METHODS FOR MODIFYING NUCLEIC ACIDS
EP4404942A4 (en) * 2021-09-26 2026-06-17 Wave Life Sciences Ltd Compositions for the Editing of Transcripts MECP2 and Related Processes
WO2023140694A1 (ko) * 2022-01-24 2023-07-27 주식회사 툴젠 스트렙토코커스 피오게네스 유래 cas9 변이체
CA3266991A1 (en) * 2022-09-08 2024-03-14 The University Court Of The University Of Edinburgh TREATMENT OF RETT SYNDROME
WO2024102811A2 (en) * 2022-11-08 2024-05-16 The Board Of Trustees Of The Leland Stanford Junior University Muscle-specific base editors for correction of mutations causing- dilated cardiomyopathy
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
JP2026524490A (ja) * 2023-07-07 2026-07-22 ミリアド ジェネティクス，インコーポレーテッド 乳がんについての多祖先ポリジェニックリスク評価
WO2025199339A2 (en) * 2024-03-20 2025-09-25 Shape Therapeutics Inc. Mecp2-targeting engineered guide rnas and polynucleotides

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019078263A1 (ja) 2017-10-17 2019-04-25 国立大学法人京都大学 多能性幹細胞から人工神経筋接合部を得る方法

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201510327TA (en) * 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
KR20250012194A (ko) * 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
EP3957731A1 (en) * 2015-07-15 2022-02-23 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
WO2018027078A1 (en) * 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
RU2019133002A (ru) * 2017-03-24 2021-04-26 Зе Юнивёрсити Корт Оф Зе Юнивёрсити Оф Эдинбург ЭКСПРЕССИОННЫЕ КАССЕТЫ MeCP2
IL316453A (en) * 2017-10-06 2024-12-01 Univ Oregon Health & Science RNA editing agents and methods
CA3082251A1 (en) * 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
KR102853088B1 (ko) * 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019078263A1 (ja) 2017-10-17 2019-04-25 国立大学法人京都大学 多能性幹細胞から人工神経筋接合部を得る方法

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JP2022533673A (ja) 2022-07-25
KR20220010540A (ko) 2022-01-25
CN114206395A (zh) 2022-03-18
CN120346348A (zh) 2025-07-22
WO2020236936A1 (en) 2020-11-26
AU2020279751A1 (en) 2021-12-02
EP3972654A4 (en) 2024-07-03
CA3140093A1 (en) 2020-11-26
US20220387622A1 (en) 2022-12-08
CN114206395B (zh) 2025-03-28
EP3972654A1 (en) 2022-03-30

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