CN116507730A - 封闭端dna载体及其用于表达苯丙氨酸羟化酶（pah）的用途 - Google Patents

封闭端dna载体及其用于表达苯丙氨酸羟化酶（pah）的用途 Download PDF

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CN116507730A
CN116507730A CN202180076781.0A CN202180076781A CN116507730A CN 116507730 A CN116507730 A CN 116507730A CN 202180076781 A CN202180076781 A CN 202180076781A CN 116507730 A CN116507730 A CN 116507730A
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seq
nucleic acid
vector
acid sequence
cenna
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Chinese (zh)
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L·Y·刘
R·蒙茲
M·J·基科
N·西尔弗
P·萨马约亚
D·A·科尔
J·L·基南
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Generational Biology Co
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Generational Biology Co
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CN202180076781.0A 2020-09-16 2021-09-16 封闭端dna载体及其用于表达苯丙氨酸羟化酶（pah）的用途 Pending CN116507730A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063078954P 2020-09-16 2020-09-16
US63/078,954 2020-09-16
PCT/US2021/050695 WO2022061000A1 (en) 2020-09-16 2021-09-16 Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah)

Publications (1)

Publication Number Publication Date
CN116507730A true CN116507730A (zh) 2023-07-28

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CN202180076781.0A Pending CN116507730A (zh) 2020-09-16 2021-09-16 封闭端dna载体及其用于表达苯丙氨酸羟化酶（pah）的用途

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US (1) US20240026374A1 (https=)
EP (1) EP4214309A4 (https=)
JP (1) JP2023542131A (https=)
KR (1) KR20230066615A (https=)
CN (1) CN116507730A (https=)
AU (1) AU2021345187A1 (https=)
CA (1) CA3191765A1 (https=)
IL (1) IL301196A (https=)
MX (1) MX2023003019A (https=)
WO (1) WO2022061000A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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.
GB202307239D0 (en) * 2023-05-15 2023-06-28 Evox Therapeutics Ltd Gene therapies for Phenylketonuria
WO2025064507A1 (en) * 2023-09-19 2025-03-27 Poseida Therapeutics, Inc. Compositions and methods for integration of viral vectors

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11007280B2 (en) * 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
US12442015B2 (en) * 2018-01-19 2025-10-14 Generation Bio Co. Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
MA51915A (fr) * 2018-02-22 2020-12-30 Generation Bio Co Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna)
TW202016314A (zh) * 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
CN113454232B (zh) * 2019-02-15 2024-10-29 世代生物公司 封闭端dna（cedna）产生中对rep蛋白活性的调节
EP3938515A4 (en) * 2019-03-13 2022-12-28 Generation Bio Co. NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS
BR112021024124A2 (pt) * 2019-05-31 2022-01-11 American Gene Tech Int Inc Expressão otimizada de fenilalanina hidroxilase

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US20240026374A1 (en) 2024-01-25
EP4214309A1 (en) 2023-07-26
IL301196A (en) 2023-05-01
KR20230066615A (ko) 2023-05-16
AU2021345187A1 (en) 2023-04-06
MX2023003019A (es) 2023-05-08
EP4214309A4 (en) 2025-07-16
WO2022061000A1 (en) 2022-03-24
CA3191765A1 (en) 2022-03-24
JP2023542131A (ja) 2023-10-05

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