JP7586601B2 - 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 - Google Patents

遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 Download PDF

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JP7586601B2
JP7586601B2 JP2021546888A JP2021546888A JP7586601B2 JP 7586601 B2 JP7586601 B2 JP 7586601B2 JP 2021546888 A JP2021546888 A JP 2021546888A JP 2021546888 A JP2021546888 A JP 2021546888A JP 7586601 B2 JP7586601 B2 JP 7586601B2
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gene
base editor
regulatory element
nucleic acid
adenosine
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JP2022520080A (ja
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イアン スレイメイカー、
ニコル ゴーデッリ、
イ ユ、
ベルンド ゼッツェ、
デイビッド エー． ボーン、
スン―ジュ イ、
マイケル パッカー、
ジェイソン マイケル ゲールケ、
ナタリー ペトロシャン、
アンジェリカ メッサーナ、
シャウナ バーコヴィッチ、
デイビッド ブライソン、
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ビーム セラピューティクス インク．
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JP2021546888A 2019-02-13 2020-02-13 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 Active JP7586601B2 (ja)

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JP2024193416A JP2025032080A (ja) 2019-02-13 2024-11-05 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法

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Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962850919P 2019-05-21 2019-05-21
US62/850,919 2019-05-21
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,224 2019-05-23
US62/852,228 2019-05-23
US201962873138P 2019-07-11 2019-07-11
US62/873,138 2019-07-11
US201962888867P 2019-08-19 2019-08-19
US62/888,867 2019-08-19
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018073 WO2020168051A1 (en) 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

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JP2022520080A JP2022520080A (ja) 2022-03-28
JPWO2020168051A5 JPWO2020168051A5 (enExample) 2023-02-21
JP7586601B2 true JP7586601B2 (ja) 2024-11-19

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JP2024193416A Pending JP2025032080A (ja) 2019-02-13 2024-11-05 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法

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JP (2) JP7586601B2 (enExample)
KR (1) KR20210127206A (enExample)
CN (2) CN114040970B (enExample)
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JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
CN116497067B (zh) 2019-02-13 2025-01-24 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
CR20210572A (es) 2019-04-15 2022-04-07 Univ Beijing Métodos y composiciones para editar ácido ribonucleicos (arn)
PH12022550059A1 (en) 2019-07-12 2022-11-21 Univ Beijing Targeted rna editing by leveraging endogenous adar using engineered rnas
AU2020418228A1 (en) * 2019-12-30 2022-08-18 Edigene Therapeutics (Beijing) Inc. Leaper technology based method for treating MPS IH and composition
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WO2022027035A1 (en) * 2020-07-27 2022-02-03 The Children's Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
EP4232584A4 (en) * 2020-10-21 2025-07-16 Univ Leland Stanford Junior SCREENING PLATFORM FOR RECRUITING GUIDE RNAS
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EP4441219A2 (en) * 2021-12-01 2024-10-09 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides for rna editing targeting lrrk2
CN117187220A (zh) * 2022-03-08 2023-12-08 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
CN114686456B (zh) 2022-05-10 2023-02-17 中山大学 基于双分子脱氨酶互补的碱基编辑系统及其应用
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CN121152875A (zh) * 2023-05-09 2025-12-16 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
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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
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