The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
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DOI：
10.1038/s41565-019-0591-y
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发表时间：
2019-12-01
影响因子：
38.3
通讯作者：
Cullis, Pieter R.
中科院分区：
文献类型：
--
作者：
Akinc, Akin;Maier, Martin A.;Cullis, Pieter R.
Onpattro是一种基于脂质纳米颗粒的短干扰RNA药物，用于治疗遗传性甲状腺素运载蛋白淀粉样变性诱导的多发性神经病，其监管批准为许多基于核酸的疗法的临床开发铺平了道路。
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.