CN111433368A - 具有启动子和增强子组合的载体用于治疗苯丙酮尿症 - Google Patents

具有启动子和增强子组合的载体用于治疗苯丙酮尿症 Download PDF

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CN111433368A
CN111433368A CN201880077904.0A CN201880077904A CN111433368A CN 111433368 A CN111433368 A CN 111433368A CN 201880077904 A CN201880077904 A CN 201880077904A CN 111433368 A CN111433368 A CN 111433368A
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pah
sequence
seq
vector
promoter
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T·拉胡森
C·D·保扎
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American Gene Technologies International Inc
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American Gene Technologies International Inc
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CN201880077904.0A 2017-10-02 2018-10-02 具有启动子和增强子组合的载体用于治疗苯丙酮尿症 Pending CN111433368A (zh)

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US201762566979P 2017-10-02 2017-10-02
US62/566,979 2017-10-02
PCT/US2018/053919 WO2019070674A2 (en) 2017-10-02 2018-10-02 VECTORS COMPRISING PROMOTER AND ACTIVATOR COMBINATIONS FOR THE TREATMENT OF PHENYLCETONURIA

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CN111433368A true CN111433368A (zh) 2020-07-17

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US (1) US20200318081A1 (enExample)
EP (1) EP3692157A4 (enExample)
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CN (1) CN111433368A (enExample)
AU (1) AU2018345745A1 (enExample)
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JP7173548B2 (ja) 2016-06-08 2022-11-16 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 非組み込みウイルス送達系およびその関連方法
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CA3165015A1 (en) * 2020-01-16 2021-07-22 Matthias Klugmann Adeno associated virus based gene therapy for phenylketonuria
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
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