JP7631209B2 - 糖原病１ａ型を治療するための組成物および方法 - Google Patents

糖原病１ａ型を治療するための組成物および方法 Download PDF

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JP7631209B2
JP7631209B2 JP2021546892A JP2021546892A JP7631209B2 JP 7631209 B2 JP7631209 B2 JP 7631209B2 JP 2021546892 A JP2021546892 A JP 2021546892A JP 2021546892 A JP2021546892 A JP 2021546892A JP 7631209 B2 JP7631209 B2 JP 7631209B2
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amino acid
cas9
domain
tada
adenosine deaminase
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JP2022519882A5 (https=
JP2022519882A (ja
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ニコル ゴーデッリ、
マイケル パッカー、
イアン スレイメイカー、
イ ユ、
ベルンド ゼッツェ、
イボンヌ アラティン、
フランシーヌ グレゴワール、
ジェネシス ラング、
デイビッド エー． ボーン、
スン―ジュ イ、
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ビーム セラピューティクス インク．
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JP2021546892A 2019-02-13 2020-02-13 糖原病１ａ型を治療するための組成物および方法 Active JP7631209B2 (ja)

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US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962852228P 2019-05-23 2019-05-23
US201962852224P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 2019-05-23
US201962876354P 2019-07-19 2019-07-19
US62/876,354 2019-07-19
US201962912992P 2019-10-09 2019-10-09
US62/912,992 2019-10-09
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/US2020/018124 WO2020168088A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating glycogen storage disease type 1a

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JP2025087691A (ja) * 2019-02-13 2025-06-10 ビーム セラピューティクス インク． 糖原病１ａ型を治療するための組成物および方法

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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
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
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
JP2023505234A (ja) * 2019-12-04 2023-02-08 アーバー バイオテクノロジーズ， インコーポレイテッド ヌクレアーゼを含む組成物及びその使用
US12416001B2 (en) * 2020-02-14 2025-09-16 Ohio State Innovation Foundation Nucleobase editors and methods of use thereof
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