JP2021512871A - Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びそれらの使用方法 - Google Patents

Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びそれらの使用方法 Download PDF

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JP2021512871A
JP2021512871A JP2020541887A JP2020541887A JP2021512871A JP 2021512871 A JP2021512871 A JP 2021512871A JP 2020541887 A JP2020541887 A JP 2020541887A JP 2020541887 A JP2020541887 A JP 2020541887A JP 2021512871 A JP2021512871 A JP 2021512871A
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amino acid
seq
capsid protein
pah
nucleotide
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JP2021512871A5 (OSRAM
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アルバート・バーンズ・シーモア
シーミン・セヘル・アーメド
ジェーソン・ボーク・ライト
セリーナ・ニコル・ドリヴ
ジェームズ・アンソニー・マクスウィゲン
ジェイミー・ミッシェル・プラウト
ダニエル・ローレン・ソッキアシアン
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ホモロジー・メディシンズ・インコーポレイテッド
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JP2020541887A 2018-02-01 2019-02-01 Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びそれらの使用方法 Ceased JP2021512871A (ja)

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US201862625149P 2018-02-01 2018-02-01
US62/625,149 2018-02-01
US201862672377P 2018-05-16 2018-05-16
US62/672,377 2018-05-16
PCT/US2019/016354 WO2019152843A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

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JP2021512871A true JP2021512871A (ja) 2021-05-20
JP2021512871A5 JP2021512871A5 (OSRAM) 2022-02-09

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US (3) US20190256867A1 (OSRAM)
EP (1) EP3746101A4 (OSRAM)
JP (1) JP2021512871A (OSRAM)
KR (1) KR20200127170A (OSRAM)
CN (1) CN111902164A (OSRAM)
AU (1) AU2019215152A1 (OSRAM)
BR (1) BR112020015798A2 (OSRAM)
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CO (1) CO2020010793A2 (OSRAM)
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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
MX2022008677A (es) * 2020-01-16 2022-08-10 Takeda Pharmaceuticals Co Terapia genica basada en virus adenoasociados para la fenilcetonuria.
US20210361778A1 (en) * 2020-04-06 2021-11-25 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
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
US12173308B2 (en) 2020-10-01 2024-12-24 Genzyme Corporation Human PAH expression cassette for treatment of PKU by liver-directed gene replacement therapy

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