小干扰RNA
体内
RNA干扰
结合
药理学
计算生物学
药物输送
全身给药
化学
核糖核酸
医学
生物
基因
生物化学
生物技术
数学分析
有机化学
数学
作者
Maire F. Osborn,Anastasia Khvorova
出处
期刊:Nucleic Acid Therapeutics
[Mary Ann Liebert]
日期:2018-06-01
卷期号:28 (3): 128-136
被引量:91
标识
DOI:10.1089/nat.2018.0725
摘要
RNA interference (RNAi)-based therapeutics are approaching clinical approval for genetically defined diseases. Current clinical success is a result of significant innovations in the development of chemical architectures that support sustained, multi-month efficacy in vivo following a single administration. Conjugate-mediated delivery has established itself as the most promising platform for safe and targeted small interfering RNA (siRNA) delivery. Lipophilic conjugates represent a major class of modifications that improve siRNA pharmacokinetics and enable efficacy in a broad range of tissues. Here, we review current literature and define key features and limitations of this approach for in vivo modulation of gene expression.
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