Delivery of oligonucleotides with lipid nanoparticles.
Delivery of oligonucleotides with lipid nanoparticles.
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DOI：
10.1016/j.addr.2015.02.007
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发表时间：
2015-06-29
影响因子：
16.1
通讯作者：
Huang L
中科院分区：
文献类型：
--
作者：
Wang Y;Miao L;Satterlee A;Huang L
自 20 世纪 80 年代问世以来，基于寡核苷酸（ON-based）的疗法已被认为是可以治疗多种疾病的强大工具。具有不同作用机制的新型基因表达调控方法的发现仍在推动新型基于 ON 的疗法的发展。此类疗法的递送困难阻碍了它们的体内应用，这迫使药物递送系统成为临床转化的先决条件。本综述讨论了使用脂质纳米颗粒作为载体在体外和体内将治疗性 ON 递送至靶细胞的策略。关于如何在配制过程中利用脂质材料的化学和物理特性以及由此对递送效率产生的影响的讨论构成了本综述的主要部分。
Since their inception in the 1980's, oligonucleotide-based (ON-based) therapeutics have been recognized as powerful tools that can treat a broad spectrum of diseases. The discoveries of novel regulatory methods of gene expression with diverse mechanisms of action are still driving the development of novel ON-based therapeutics. Difficulties in the delivery of this class of therapeutics hinder their in vivo applications, which forces drug delivery systems to be a prerequisite for clinical translation. This review discusses the strategy of using lipid nanoparticles as carriers to deliver therapeutic ONs to target cells in vitro and in vivo. A discourse on how chemical and physical properties of the lipid materials could be utilized during formulation and the resulting effects on delivery efficiency constitutes the major part of this review.