核酸治疗的靶向性传递

Targeted Delivery for Oligonucleotide Molecular Therapeutics

寡核苷酸包括反义核酸和干扰RNA等,它们能够为多种疾病提供快速、特异性的治疗,具有很高的应用潜力。然而这些分子能否有效地传递到特定的细胞和组织对于最终的临床应用非常关键。靶向性的传递能够提高寡核苷酸药物的特异性和使用效率,使药物分子能有效到达作用位点,进而发挥它们的治疗效果。一些基于不同平台的靶向性配体传递策略已经形成,并能够很好的发挥其生物学活性。本文针对当前该研究领域的进展提供一个概述。

Oligonucleotides including antisense oligonucleotides and small interfering RNA can provide for a variety diseases,rapid,specific treatment,with a high potential for application.However,effective delivery of oligonucleotide molecules to specific cells and tissues is critical for finally clinical application.Targeted system can greatly improve the efficiency and specificity of oligonucleotides delivery.Meanwhile,an effective delivery system enable the oligonucleotides to reach the sites of action and access their biological targets.Some delivery strategies based on different platforms and different targeting ligands have been developed,and can play their biological activity.In this paper,the current progress in the field of the study will be summarized.