KR20220010540A - 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법 - Google Patents

프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법 Download PDF

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KR20220010540A
KR20220010540A KR1020217041287A KR20217041287A KR20220010540A KR 20220010540 A KR20220010540 A KR 20220010540A KR 1020217041287 A KR1020217041287 A KR 1020217041287A KR 20217041287 A KR20217041287 A KR 20217041287A KR 20220010540 A KR20220010540 A KR 20220010540A
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tada
domain
rett
amino acid
adenosine deaminase
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제이슨 마이클 게르케
나탈리 페트로시안
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빔 테라퓨틱스, 인크.
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KR1020217041287A 2019-05-21 2020-05-20 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법 Pending KR20220010540A (ko)

Applications Claiming Priority (3)

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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KR20220010540A true KR20220010540A (ko) 2022-01-25

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KR1020217041287A Pending KR20220010540A (ko) 2019-05-21 2020-05-20 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법

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US (1) US20220387622A1 (https=)
EP (1) EP3972654A4 (https=)
JP (1) JP7679311B2 (https=)
KR (1) KR20220010540A (https=)
CN (2) CN120346348A (https=)
AU (1) AU2020279751A1 (https=)
CA (1) CA3140093A1 (https=)
WO (1) WO2020236936A1 (https=)

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WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
AU2019265019B2 (en) 2018-05-11 2025-11-06 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
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
JP7693552B2 (ja) * 2019-02-13 2025-06-17 ビーム セラピューティクス インク． アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20240352439A1 (en) * 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
EP4404942A2 (en) * 2021-09-26 2024-07-31 Wave Life Sciences Ltd. Compositions for editing mecp2 transcripts and methods thereof
US20250154479A1 (en) * 2022-01-24 2025-05-15 Toolgen Incorporated Streptococcus pyogenes-derived cas9 variant
JP2025530183A (ja) * 2022-09-08 2025-09-11 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ レット症候群治療法
EP4615574A2 (en) * 2022-11-08 2025-09-17 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
WO2025014806A1 (en) * 2023-07-07 2025-01-16 Myriad Genetics, Inc. Multiple-ancestry polygenic risk assessment for breast cancer
WO2025199339A2 (en) * 2024-03-20 2025-09-25 Shape Therapeutics Inc. Mecp2-targeting engineered guide rnas and polynucleotides

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MX374532B (es) * 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
CN105683379A (zh) * 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
JP7044373B2 (ja) * 2015-07-15 2022-03-30 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
CN110214183A (zh) * 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
BR112019019833A2 (pt) * 2017-03-24 2020-04-22 The University Court Of The University Of Edinburgh terapia baseada em mecp2
WO2019071274A1 (en) * 2017-10-06 2019-04-11 Oregon Health & Science University Compositions and methods for editing rna
KR20250107288A (ko) * 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3699267A4 (en) 2017-10-17 2021-10-27 Kyoto University PROCESS FOR OBTAINING AN ARTIFICIAL NEUROMUSCULAR JUNCTION FROM PLURIPOTENT STEM CELLS
KR20250133482A (ko) * 2018-05-11 2025-09-05 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법

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JP7679311B2 (ja) 2025-05-19
JP2022533673A (ja) 2022-07-25
CN114206395B (zh) 2025-03-28
CA3140093A1 (en) 2020-11-26
EP3972654A1 (en) 2022-03-30
US20220387622A1 (en) 2022-12-08
AU2020279751A1 (en) 2021-12-02
WO2020236936A1 (en) 2020-11-26
EP3972654A4 (en) 2024-07-03
CN114206395A (zh) 2022-03-18
CN120346348A (zh) 2025-07-22

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