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
JPWO2020168051A5 (https=
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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

Applications Claiming Priority (19)

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
US62/852,228 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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JP2024193416A Division 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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JP2022520080A JP2022520080A (ja) 2022-03-28
JPWO2020168051A5 JPWO2020168051A5 (https=) 2023-02-21
JP2022520080A5 JP2022520080A5 (https=) 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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US (1) US20230140953A1 (https=)
EP (1) EP3924484A4 (https=)
JP (2) JP7586601B2 (https=)
KR (1) KR20210127206A (https=)
CN (2) CN119280261A (https=)
AU (1) AU2020223306A1 (https=)
CA (1) CA3128876A1 (https=)
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KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
KR102851101B1 (ko) 2019-04-15 2025-08-27 에디진 테라퓨틱스(베이징) 인크. Rna를 편집하기 위한 방법 및 조성물
PE20220300A1 (es) 2019-07-12 2022-03-09 Univ Beijing Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente
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US20230140953A1 (en) 2023-05-11
AU2020223306A1 (en) 2021-08-05
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WO2020168051A9 (en) 2020-10-22
CA3128876A1 (en) 2020-08-20
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