JP2021500072A5 - - Google Patents

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JP2021500072A5
JP2021500072A5 JP2020542542A JP2020542542A JP2021500072A5 JP 2021500072 A5 JP2021500072 A5 JP 2021500072A5 JP 2020542542 A JP2020542542 A JP 2020542542A JP 2020542542 A JP2020542542 A JP 2020542542A JP 2021500072 A5 JP2021500072 A5 JP 2021500072A5
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nucleic acid
grna
dna endonuclease
dna
acid encoding
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JP2020542542A
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Japanese (ja)
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JP7482028B2 (ja
JP2021500072A (ja
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Priority claimed from PCT/US2018/056390 external-priority patent/WO2019079527A1/en
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Priority to JP2023188673A priority Critical patent/JP2024009014A/ja
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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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023188673A JP2024009014A (ja) 2017-10-17 2023-11-02 血友病ａに対する遺伝子編集用組成物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762573633P 2017-10-17 2017-10-17
US62/573,633 2017-10-17
PCT/US2018/056390 WO2019079527A1 (en) 2017-10-17 2018-10-17 COMPOSITIONS AND METHODS FOR GENETIC EDITION FOR HEMOPHILIA A

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JP2021500072A JP2021500072A (ja) 2021-01-07
JP2021500072A5 true JP2021500072A5 (enExample) 2021-11-25
JP7482028B2 JP7482028B2 (ja) 2024-05-13

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JP2020542542A Active JP7482028B2 (ja) 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
JP2023188673A Pending JP2024009014A (ja) 2017-10-17 2023-11-02 血友病ａに対する遺伝子編集用組成物及び方法

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US (3) US20190247517A1 (enExample)
EP (1) EP3697907A1 (enExample)
JP (2) JP7482028B2 (enExample)
KR (1) KR102728416B1 (enExample)
CN (1) CN111684070A (enExample)
AU (1) AU2018353012B2 (enExample)
BR (1) BR112020007502A2 (enExample)
CA (1) CA3079172A1 (enExample)
IL (1) IL273999A (enExample)
MA (1) MA50833A (enExample)
MX (1) MX2020004043A (enExample)
SG (1) SG11202003464VA (enExample)
WO (1) WO2019079527A1 (enExample)

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MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
BR112021015776A2 (pt) * 2019-06-05 2021-11-09 Bayer Healthcare Llc Edição de gene para hemofilia a com expressão de fator viii melhorada
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
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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
WO2021091927A1 (en) * 2019-11-04 2021-05-14 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
WO2023172875A1 (en) * 2022-03-07 2023-09-14 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
EP4514837A1 (en) * 2022-04-25 2025-03-05 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
EP4574975A1 (en) * 2023-12-20 2025-06-25 Bayer AG Compositions and methods for delivering transgenes
WO2025153529A1 (en) * 2024-01-16 2025-07-24 Novo Nordisk A/S Adeno-associated virus vector sequence

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