JP2022535745A5 - - Google Patents

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Publication number
JP2022535745A5
JP2022535745A5 JP2021570364A JP2021570364A JP2022535745A5 JP 2022535745 A5 JP2022535745 A5 JP 2022535745A5 JP 2021570364 A JP2021570364 A JP 2021570364A JP 2021570364 A JP2021570364 A JP 2021570364A JP 2022535745 A5 JP2022535745 A5 JP 2022535745A5
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JP
Japan
Prior art keywords
sequence
seq
percent
codon
viral vector
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
JP2021570364A
Other languages
English (en)
Japanese (ja)
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JP2022535745A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/035584 external-priority patent/WO2020243717A1/en
Publication of JP2022535745A publication Critical patent/JP2022535745A/ja
Publication of JP2022535745A5 publication Critical patent/JP2022535745A5/ja
Pending legal-status Critical Current

Links

JP2021570364A 2019-05-31 2020-06-01 最適化されたフェニルアラニンヒドロキシラーゼ発現 Pending JP2022535745A (ja)

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 (2)

Publication Number Publication Date
JP2022535745A JP2022535745A (ja) 2022-08-10
JP2022535745A5 true JP2022535745A5 (enExample) 2023-06-08

Family

ID=73553945

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021570364A Pending JP2022535745A (ja) 2019-05-31 2020-06-01 最適化されたフェニルアラニンヒドロキシラーゼ発現

Country Status (10)

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

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
CN116064678A (zh) 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. SYSTEM FOR THE NON-INTEGRAL VIRAL ISSUE AND PROCEDURE RELATING TO IT
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR THE TREATMENT OF PARKINSON'S DISEASE
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
JP2020524996A (ja) 2017-06-16 2020-08-27 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 ヒトガンマデルタt細胞を介する腫瘍細胞傷害性の活性化のための方法および組成物
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
MX2024003186A (es) * 2021-09-16 2024-04-23 Generation Bio Co Casetes de expresión específicos del hígado, vectores y usos de estos para expresar proteínas terapéuticas.
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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
JP6506749B2 (ja) * 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのmRNA療法
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
US20180363000A1 (en) * 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
EP3825400B1 (en) * 2016-04-08 2024-12-25 Translate Bio, 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
US11820999B2 (en) * 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
CA3077355A1 (en) * 2017-10-02 2019-04-11 American Gene Technologies International Inc. Vectors with promoter and enhancer combinations for treating phenylketonuria

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