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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amino acid
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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    • 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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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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 (https=) 2023-05-29
JP7679311B2 true JP7679311B2 (ja) 2025-05-19

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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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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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BR112019019833A2 (pt) * 2017-03-24 2020-04-22 The University Court Of The University Of Edinburgh terapia baseada em mecp2
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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
KR20220010540A (ko) 2022-01-25
EP3972654A4 (en) 2024-07-03
CN114206395A (zh) 2022-03-18
CN120346348A (zh) 2025-07-22

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