核糖核酸
寡核苷酸
遗传增强
基因传递
纳米医学
纳米技术
药物输送
计算生物学
生物
基因
纳米颗粒
遗传学
材料科学
作者
Keith Henry Moss,Petya Popova,Sine Reker Hadrup,Kira Astakhova,Maria Taskova
标识
DOI:10.1021/acs.molpharmaceut.8b01290
摘要
Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.
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