JP2022504740A - 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成 - Google Patents

肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成 Download PDF

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JP2022504740A
JP2022504740A JP2021519879A JP2021519879A JP2022504740A JP 2022504740 A JP2022504740 A JP 2022504740A JP 2021519879 A JP2021519879 A JP 2021519879A JP 2021519879 A JP2021519879 A JP 2021519879A JP 2022504740 A JP2022504740 A JP 2022504740A
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nucleic acid
promoter
pah polypeptide
raav
aav
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JP2022504740A5 (https=
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シルッカ・キョスティオ－ムーア
パルタ・マナヴァラン
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Genzyme Corp
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Genzyme Corp
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JP2021519879A 2018-10-12 2019-10-11 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成 Pending JP2022504740A (ja)

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JP2024098655A JP2024123125A (ja) 2018-10-12 2024-06-19 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成

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US201862744944P 2018-10-12 2018-10-12
US62/744,944 2018-10-12
PCT/US2019/055917 WO2020077250A1 (en) 2018-10-12 2019-10-11 Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy

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JP2024098655A Division JP2024123125A (ja) 2018-10-12 2024-06-19 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成

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JP2024098655A Pending JP2024123125A (ja) 2018-10-12 2024-06-19 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成

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JP (2) JP2022504740A (https=)
KR (1) KR20210076051A (https=)
CN (2) CN120349985A (https=)
AR (1) AR116700A1 (https=)
AU (2) AU2019357039B2 (https=)
BR (1) BR112021006796A2 (https=)
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IL (1) IL282140A (https=)
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EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
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AU2021261359A1 (en) * 2020-04-21 2022-12-08 Allen Institute Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons
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TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
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AU2022332276A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
CN115572730A (zh) * 2022-04-24 2023-01-06 复旦大学附属中山医院 一种gtf2h4高表达的内皮细胞系
CN115772503B (zh) * 2022-08-16 2023-10-17 广东省南山医药创新研究院 一种表达pah的基因修饰细胞药物及其制备方法与应用
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
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AU2019357039A1 (en) 2021-06-03
CN120349985A (zh) 2025-07-22
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MX2025013445A (es) 2025-12-01
KR20210076051A (ko) 2021-06-23
AU2025237999A1 (en) 2025-10-23
EP4461814A2 (en) 2024-11-13
TW202035439A (zh) 2020-10-01
SG11202103614RA (en) 2021-05-28
PH12021550794A1 (en) 2021-12-13
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CA3116283A1 (en) 2020-04-16
CN113194982A (zh) 2021-07-30
EP3863662A1 (en) 2021-08-18
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AU2019357039B2 (en) 2025-10-09
JP2024123125A (ja) 2024-09-10
IL282140A (en) 2021-05-31
AR116700A1 (es) 2021-06-02
WO2020077250A1 (en) 2020-04-16
CO2021006060A2 (es) 2021-05-20
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