TWI875705B - 核酸分子及其用於非病毒基因療法的用途 - Google Patents

核酸分子及其用於非病毒基因療法的用途 Download PDF

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TWI875705B
TWI875705B TW108128641A TW108128641A TWI875705B TW I875705 B TWI875705 B TW I875705B TW 108128641 A TW108128641 A TW 108128641A TW 108128641 A TW108128641 A TW 108128641A TW I875705 B TWI875705 B TW I875705B
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itr
nucleic acid
seq
sequence
nucleotides
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TW108128641A
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TW202023630A (zh
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彤瑤 劉
阿列希 賽瑞吉
羅伯特 彼得斯
劉佳云
飛利浦 查卡斯
道格拉斯 查格
懷特 蘇珊娜 帕塔羅約
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美商百歐維拉提夫治療公司
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    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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TW108128641A 2018-08-09 2019-08-12 核酸分子及其用於非病毒基因療法的用途 TWI875705B (zh)

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US (2) US12364774B2 (https=)
EP (1) EP3833766A1 (https=)
JP (1) JP7602454B2 (https=)
KR (2) KR102925832B1 (https=)
CN (2) CN113227385B (https=)
AU (1) AU2019319984B2 (https=)
BR (1) BR112021002017A2 (https=)
CA (1) CA3108799A1 (https=)
CO (1) CO2021002965A2 (https=)
IL (1) IL280652A (https=)
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NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
AU2019375450A1 (en) * 2018-11-07 2021-06-17 Vivet Therapeutics Codon-optimized ABCB11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (PFIC2)
CN111334528B (zh) * 2020-03-20 2022-02-18 山东农业大学 水貂肠炎细小病毒全基因组感染性克隆及其构建方法和应用
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Dna分子の組成物、その作製方法、及びその使用方法
WO2022046665A1 (en) * 2020-08-23 2022-03-03 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CN112704729A (zh) * 2020-12-28 2021-04-27 江苏纳迪芯生命科技研究院有限公司 一种细胞因子颗粒
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
CN113325181A (zh) * 2021-04-25 2021-08-31 苏州市立医院(北区) 丝氨酸蛋白酶抑制剂在用于脓毒症早期预警的标志物中的应用
US20240209393A1 (en) * 2021-04-26 2024-06-27 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
US20240287493A1 (en) * 2021-06-24 2024-08-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
MX2024002333A (es) 2021-08-23 2024-03-07 Bioverativ Therapeutics Inc Sistema de expresion de baculovirus.
AU2022332276A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
JP2024532261A (ja) 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 操作itr配列および使用方法
CN115820740A (zh) * 2021-09-16 2023-03-21 北京锦篮基因科技有限公司 用于治疗ⅱ型粘多糖贮积症的重组腺相关病毒载体及其应用
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