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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JP2022520080A5 (enExample
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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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
US62/852,228 2019-05-23
US62/852,224 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
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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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EP (1) EP3924484A4 (enExample)
JP (2) JP7586601B2 (enExample)
KR (1) KR20210127206A (enExample)
CN (2) CN119280261A (enExample)
AU (1) AU2020223306A1 (enExample)
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AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
CN114040977B (zh) 2019-01-31 2024-09-03 比姆医疗股份有限公司 非靶脱氨反应减低的核碱基编辑器和用于定性核碱基编辑器的测定
CA3236512C (en) 2019-02-13 2026-02-24 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
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SG11202108346WA (en) 2019-02-13 2021-08-30 Beam Therapeutics Inc Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
PH12021552613A1 (en) 2019-04-15 2022-06-27 Edigene Therapeutics Beijing Inc METHODS AND COMPOSITIONS FOR EDITING RNAs
CN114269919B (zh) 2019-07-12 2023-08-18 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑
CN115279398B (zh) 2019-09-27 2025-07-04 比姆医疗股份有限公司 治疗液态癌症的组合物和方法
PH12022551595A1 (en) * 2019-12-30 2023-11-29 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
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