【摘要】 小RNA药物应用于临床的主要技术瓶颈在于如何高效、低毒地将小RNA分子传递到它发挥功能的场所.基于细胞穿透肽在小RNA透皮给药的临床应用中所取得的进展,本文系统评述了近年来细胞穿透肽在小RNA的体内、体外传递方面的研究动态,分析了细胞穿透肽的结构改造对肽/小RNA复合物转染进入细胞发挥功能的影响,展望了细胞穿透肽作为小RNA的体内药物传递载体的发展方向.更多还原

【Abstract】 The less of high efficient carriers to deliver siRNA drugs into their targeted tissues/cells with less toxicity are the major obstacles of siRNA pharmaceuticals in clinic application.The recent advance of siRNA delivery research based on cell-penetrating peptides(CPPs) in clinic opened the way of siRNA techniques for therapeutic application(Yi et al.,Mol Ther(2011)19,362-371).CPPs are short amphipathic and cationic peptides that are rapidly internalized across cell membranes.They can be used to deliver molecular cargos,such as imaging agents(fluorescent dyes and quantum dots),drugs,liposomes,peptide/protein,oligonucleotide/DNA/RNA,nanoparticles and bacteriophage into cells.The mechanism of cellular uptake and subsequent processing still remains controversial.It is now clear that CPP can mediate intracellular delivery via both endocytic and non-endocytic pathways.In this review,we discuss potential functions of CPPs,especially in structure modified CPPs for small RNA delivery in vitro and in vivo,highlighting their powerful promise for clinical efficacy.更多还原