JP2022504740A5 - - Google Patents

Info

Publication number
JP2022504740A5
JP2022504740A5 JP2021519879A JP2021519879A JP2022504740A5 JP 2022504740 A5 JP2022504740 A5 JP 2022504740A5 JP 2021519879 A JP2021519879 A JP 2021519879A JP 2021519879 A JP2021519879 A JP 2021519879A JP 2022504740 A5 JP2022504740 A5 JP 2022504740A5
Authority
JP
Japan
Prior art keywords
promoter
nucleic acid
pah polypeptide
amino acid
polypeptide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021519879A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022504740A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/055917 external-priority patent/WO2020077250A1/en
Publication of JP2022504740A publication Critical patent/JP2022504740A/ja
Publication of JP2022504740A5 publication Critical patent/JP2022504740A5/ja
Priority to JP2024098655A priority Critical patent/JP2024123125A/ja
Pending legal-status Critical Current

Links

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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
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

Publications (2)

Publication Number Publication Date
JP2022504740A JP2022504740A (ja) 2022-01-13
JP2022504740A5 true JP2022504740A5 (https=) 2022-10-17

Family

ID=68426839

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021519879A Pending JP2022504740A (ja) 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
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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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

Country Status (16)

Country Link
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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EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
AU2021261359A1 (en) * 2020-04-21 2022-12-08 Allen Institute Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons
JP2023524577A (ja) * 2020-05-05 2023-06-12 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 改変アデノ随伴ウイルス５カプシドおよびその使用
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
EP4158005A1 (en) * 2020-06-01 2023-04-05 ModernaTX, Inc. Phenylalanine hydroxylase variants and uses thereof
MX2023003805A (es) 2020-10-01 2023-04-12 Genzyme Corp Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado.
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
CN117899238A (zh) * 2023-12-07 2024-04-19 哈尔滨工业大学 Pglyrp2基因的用途及其相关药物

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