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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs.
Nature Nanotechnology ( IF 38.3 ) Pub Date : 2019-12-01 , DOI: 10.1038/s41565-019-0591-y Akin Akinc 1 , Martin A Maier 1 , Muthiah Manoharan 1 , Kevin Fitzgerald 1 , Muthusamy Jayaraman 1 , Scott Barros 1 , Steven Ansell 2 , Xinyao Du 2 , Michael J Hope 2 , Thomas D Madden 2 , Barbara L Mui 2 , Sean C Semple 2 , Ying K Tam 2 , Marco Ciufolini 3 , Dominik Witzigmann 3 , Jayesh A Kulkarni 3 , Roy van der Meel 3 , Pieter R Cullis 3, 4
Nature Nanotechnology ( IF 38.3 ) Pub Date : 2019-12-01 , DOI: 10.1038/s41565-019-0591-y Akin Akinc 1 , Martin A Maier 1 , Muthiah Manoharan 1 , Kevin Fitzgerald 1 , Muthusamy Jayaraman 1 , Scott Barros 1 , Steven Ansell 2 , Xinyao Du 2 , Michael J Hope 2 , Thomas D Madden 2 , Barbara L Mui 2 , Sean C Semple 2 , Ying K Tam 2 , Marco Ciufolini 3 , Dominik Witzigmann 3 , Jayesh A Kulkarni 3 , Roy van der Meel 3 , Pieter R Cullis 3, 4
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The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therapies enabled by nanoparticle delivery.
中文翻译：
包含基于核酸的药物的纳米药物的Onpattro故事和临床翻译。
Onpattro是一种基于脂质纳米颗粒的短干扰RNA药物，用于治疗遗传性运甲状腺素蛋白淀粉样变性病引起的多发性神经病，其监管批准为通过纳米颗粒递送实现许多基于核酸的疗法的临床开发铺平了道路。
更新日期：2019-12-04
中文翻译：
包含基于核酸的药物的纳米药物的Onpattro故事和临床翻译。
Onpattro是一种基于脂质纳米颗粒的短干扰RNA药物，用于治疗遗传性运甲状腺素蛋白淀粉样变性病引起的多发性神经病，其监管批准为通过纳米颗粒递送实现许多基于核酸的疗法的临床开发铺平了道路。
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