CN115176022A - 用于遗传性血管性水肿的基于腺相关病毒载体的基因疗法 - Google Patents

用于遗传性血管性水肿的基于腺相关病毒载体的基因疗法 Download PDF

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CN115176022A
CN115176022A CN202080084617.XA CN202080084617A CN115176022A CN 115176022 A CN115176022 A CN 115176022A CN 202080084617 A CN202080084617 A CN 202080084617A CN 115176022 A CN115176022 A CN 115176022A
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raav
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sequence
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M·克鲁格曼
F·霍林
J·伦格勒
P·道拉德
F·舍夫林格
H·罗滕斯坦纳
B·刚加哈兰
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Shire Human Genetics Therapies Inc
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    • A—HUMAN NECESSITIES
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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CN202080084617.XA 2019-10-23 2020-10-23 用于遗传性血管性水肿的基于腺相关病毒载体的基因疗法 Pending CN115176022A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924877P 2019-10-23 2019-10-23
US62/924,877 2019-10-23
PCT/US2020/056984 WO2021081280A1 (en) 2019-10-23 2020-10-23 Adeno-associated virus vectors based gene therapy for hereditary angioedema

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CN115176022A true CN115176022A (zh) 2022-10-11

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US (1) US20230043051A1 (https=)
EP (1) EP4048800A1 (https=)
JP (2) JP7659550B2 (https=)
CN (1) CN115176022A (https=)
WO (1) WO2021081280A1 (https=)

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JP2023503850A (ja) * 2019-11-14 2023-02-01 バイオマリン ファーマシューティカル インコーポレイテッド 肝臓特異的遺伝子療法ベクターによる遺伝性血管浮腫の治療
WO2022130014A1 (en) * 2020-12-16 2022-06-23 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
CA3207268A1 (en) * 2021-01-27 2022-08-04 Spark Therapeutics, Inc. Compositions and methods for treating hereditary angioedema
US20240408240A1 (en) * 2021-10-01 2024-12-12 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
CN117947040A (zh) 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用

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JP7659550B2 (ja) 2025-04-09
US20230043051A1 (en) 2023-02-09
JP2022553083A (ja) 2022-12-21
WO2021081280A1 (en) 2021-04-29
EP4048800A1 (en) 2022-08-31
JP2025098221A (ja) 2025-07-01

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