TW202035439A - 生成經改良的人類ｐａｈ以藉由肝導向的基因置換療法來治療重度苯丙酮尿症（ｐｋｕ） - Google Patents

生成經改良的人類ｐａｈ以藉由肝導向的基因置換療法來治療重度苯丙酮尿症（ｐｋｕ） Download PDF

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TW202035439A
TW202035439A TW108136919A TW108136919A TW202035439A TW 202035439 A TW202035439 A TW 202035439A TW 108136919 A TW108136919 A TW 108136919A TW 108136919 A TW108136919 A TW 108136919A TW 202035439 A TW202035439 A TW 202035439A
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nucleic acid
promoter
pah polypeptide
raav
aav
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TW108136919A
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摩爾 西爾卡 寇斯蒂歐
帕薩 馬納瓦蘭
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美商健臻公司
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TW108136919A 2018-10-12 2019-10-14 生成經改良的人類ｐａｈ以藉由肝導向的基因置換療法來治療重度苯丙酮尿症（ｐｋｕ） TW202035439A (zh)

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US201862744944P 2018-10-12 2018-10-12
US62/744,944 2018-10-12

Publications (1)

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TW202035439A true TW202035439A (zh) 2020-10-01

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TW108136919A TW202035439A (zh) 2018-10-12 2019-10-14 生成經改良的人類ｐａｈ以藉由肝導向的基因置換療法來治療重度苯丙酮尿症（ｐｋｕ）

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US (1) US20210348135A1 (https=)
EP (2) EP3863662A1 (https=)
JP (2) JP2022504740A (https=)
KR (1) KR20210076051A (https=)
CN (2) CN120349985A (https=)
AR (1) AR116700A1 (https=)
AU (2) AU2019357039B2 (https=)
BR (1) BR112021006796A2 (https=)
CA (1) CA3116283A1 (https=)
CO (1) CO2021006060A2 (https=)
IL (1) IL282140A (https=)
MX (2) MX2021004213A (https=)
PH (1) PH12021550794A1 (https=)
SG (1) SG11202103614RA (https=)
TW (1) TW202035439A (https=)
WO (1) WO2020077250A1 (https=)

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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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BR112021006796A2 (pt) 2021-07-20
AU2019357039A1 (en) 2021-06-03
CN120349985A (zh) 2025-07-22
CN113194982B (zh) 2025-04-01
MX2025013445A (es) 2025-12-01
KR20210076051A (ko) 2021-06-23
AU2025237999A1 (en) 2025-10-23
EP4461814A2 (en) 2024-11-13
JP2022504740A (ja) 2022-01-13
SG11202103614RA (en) 2021-05-28
PH12021550794A1 (en) 2021-12-13
MX2021004213A (es) 2021-07-16
CA3116283A1 (en) 2020-04-16
CN113194982A (zh) 2021-07-30
EP3863662A1 (en) 2021-08-18
EP4461814A3 (en) 2025-02-26
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
US20210348135A1 (en) 2021-11-11

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