KR20220068954A - 최적화된 페닐알라닌 히드록실라아제 발현 - Google Patents

최적화된 페닐알라닌 히드록실라아제 발현 Download PDF

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KR20220068954A
KR20220068954A KR1020217042390A KR20217042390A KR20220068954A KR 20220068954 A KR20220068954 A KR 20220068954A KR 1020217042390 A KR1020217042390 A KR 1020217042390A KR 20217042390 A KR20217042390 A KR 20217042390A KR 20220068954 A KR20220068954 A KR 20220068954A
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sequence
pah
seq
codon
optimized
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Korean (ko)
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타일러 라후젠
링지 샤오
씨 데이비드 파우자
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아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드
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Publication of KR20220068954A publication Critical patent/KR20220068954A/ko
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    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020217042390A 2019-05-31 2020-06-01 최적화된 페닐알라닌 히드록실라아제 발현 Pending KR20220068954A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962855506P 2019-05-31 2019-05-31
US62/855,506 2019-05-31
PCT/US2020/035584 WO2020243717A1 (en) 2019-05-31 2020-06-01 Optimized phenylalanine hydroxylase expression

Publications (1)

Publication Number Publication Date
KR20220068954A true KR20220068954A (ko) 2022-05-26

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KR1020217042390A Pending KR20220068954A (ko) 2019-05-31 2020-06-01 최적화된 페닐알라닌 히드록실라아제 발현

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Country Link
US (1) US20220162643A1 (https=)
EP (1) EP3976076A4 (https=)
JP (1) JP2022535745A (https=)
KR (1) KR20220068954A (https=)
CN (1) CN113905768A (https=)
AU (1) AU2020283069A1 (https=)
BR (1) BR112021024124A2 (https=)
CA (1) CA3137698A1 (https=)
IL (1) IL288400A (https=)
WO (1) WO2020243717A1 (https=)

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WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
JP6890831B2 (ja) 2015-07-08 2021-06-18 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Ｈｉｖ予備免疫化および免疫療法
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
DK3402483T3 (da) 2016-01-15 2024-01-02 American Gene Tech Int Inc Fremgangsmåder og sammensætninger til aktivering af gamma-delta-T-celler
US10888613B2 (en) 2016-02-08 2021-01-12 American Gene Technologies International Inc. Method of producing cells resistant to HIV infection
DK3426777T3 (da) 2016-03-09 2022-04-25 American Gene Tech Int Inc Kombinationsvektorer og fremgangsmåder til behandling af cancer
US11976292B2 (en) 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
IL284348B2 (en) 2016-07-08 2025-06-01 American Gene Tech Int Inc Hiv pre-immunization and immunotherapy
JP7176756B2 (ja) 2016-07-21 2022-11-22 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド パーキンソン病を処置するためのウイルスベクター
CA3057142A1 (en) 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
CA3064442A1 (en) 2017-06-16 2018-12-20 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
JP7423521B2 (ja) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド
US20240026374A1 (en) * 2020-09-16 2024-01-25 Generation Bio Co. Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah)
IL311240A (en) * 2021-09-16 2024-05-01 Generation Bio Co Liver-specific expression cassettes, vectors and their uses for the expression of therapeutic proteins
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
WO2025064507A1 (en) * 2023-09-19 2025-03-27 Poseida Therapeutics, Inc. Compositions and methods for integration of viral vectors

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
EA034103B1 (ru) * 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
CN115074366A (zh) * 2015-04-16 2022-09-20 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
US20180363000A1 (en) * 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
WO2017177169A1 (en) * 2016-04-08 2017-10-12 Rana Therapeutics, Inc. Multimeric coding nucleic acid and uses thereof
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
CA3057142A1 (en) * 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
WO2019070674A2 (en) * 2017-10-02 2019-04-11 American Gene Technologies International Inc. VECTORS COMPRISING PROMOTER AND ACTIVATOR COMBINATIONS FOR THE TREATMENT OF PHENYLCETONURIA

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AU2020283069A8 (en) 2022-05-12
AU2020283069A1 (en) 2022-01-06
WO2020243717A1 (en) 2020-12-03
BR112021024124A2 (pt) 2022-01-11
JP2022535745A (ja) 2022-08-10
EP3976076A1 (en) 2022-04-06
IL288400A (en) 2022-01-01
US20220162643A1 (en) 2022-05-26
CA3137698A1 (en) 2020-12-03
CN113905768A (zh) 2022-01-07
EP3976076A4 (en) 2023-06-07

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