JP7482028B2 - 血友病ａに対する遺伝子編集用組成物及び方法 - Google Patents

血友病ａに対する遺伝子編集用組成物及び方法 Download PDF

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JP7482028B2
JP7482028B2 JP2020542542A JP2020542542A JP7482028B2 JP 7482028 B2 JP7482028 B2 JP 7482028B2 JP 2020542542 A JP2020542542 A JP 2020542542A JP 2020542542 A JP2020542542 A JP 2020542542A JP 7482028 B2 JP7482028 B2 JP 7482028B2
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リチャード ブルックス アラン
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クリスパー セラピューティクス アクチェンゲゼルシャフト
バイエル ヘルスケア リミティド ライアビリティ カンパニー
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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JP2020542542A 2017-10-17 2018-10-17 血友病ａに対する遺伝子編集用組成物及び方法 Active JP7482028B2 (ja)

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PCT/US2018/056390 WO2019079527A1 (en) 2017-10-17 2018-10-17 COMPOSITIONS AND METHODS FOR GENETIC EDITION FOR HEMOPHILIA A

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EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
BR112021015776A2 (pt) * 2019-06-05 2021-11-09 Bayer Healthcare Llc Edição de gene para hemofilia a com expressão de fator viii melhorada
MX2021015122A (es) 2019-06-07 2022-04-06 Regeneron Pharma Animales no humanos que comprenden un locus de albumina humanizado.
KR20220053671A (ko) * 2019-09-04 2022-04-29 에디진 인크. 오프 타겟 평가 기반의 유전자 편집 치료의 평가 방법
WO2021083073A1 (zh) * 2019-10-31 2021-05-06 华东师范大学 一种基于肝细胞Alb基因的疾病治疗的产品
US12139705B2 (en) * 2019-11-04 2024-11-12 California Institute Of Technology Deep learning enabled spatial optical barcodes for pooled library screens
WO2021174168A1 (en) * 2020-02-28 2021-09-02 Blueallele, Llc Methods for treating gain-of-function disorders combining gene editing and gene therapy
JP7605852B2 (ja) 2020-03-06 2024-12-24 メタゲノミ，インク． クラスｉｉのｖ型ｃｒｉｓｐｒ系
EP4490299A1 (en) * 2022-03-07 2025-01-15 Inedita Bio, Inc. Methods and compositions for intron mediated- expression of regulatory elements for trait development
CN114591957B (zh) * 2022-03-22 2024-04-26 吴文书 一种重症a型血友病动物模型的构建方法及其应用
WO2023212539A1 (en) * 2022-04-25 2023-11-02 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor viii function
WO2024254519A2 (en) * 2023-06-08 2024-12-12 Mammoth Biosciences, Inc. Compositions and methods for the modification of human genes expressed by liver cells
WO2025117858A1 (en) * 2023-11-28 2025-06-05 Metagenomi, Inc. Supplementation of liver enzyme expression
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