Division of Pharmaceutics, College of Pharmacy, The University of Texas at Austin, Texas 78712-0120, USA.
J Pharm Sci. 2012 Nov;101(11):4046-66. doi: 10.1002/jps.23300. Epub 2012 Aug 28.
RNA interference has emerged as a potentially powerful tool in the treatment of genetic and acquired diseases by delivering short interfering RNA (siRNA) or microRNA (miRNA) to target genes, resulting in their silencing. However, many physicochemical and biological barriers have to be overcome to obtain efficient in vivo delivery of siRNA and miRNA molecules to the organ/tissue of interest, thereby enabling their effective clinical therapy. This review discusses the challenges associated with the use of siRNA and miRNA and describes the nonviral delivery strategies used in overcoming these barriers. More specifically, emphasis has been placed on those technologies that have progressed to clinical trials for both local and systemic siRNA and miRNA delivery.
RNA 干扰已成为通过向靶基因递送短干扰 RNA (siRNA) 或微小 RNA (miRNA) 来治疗遗传和获得性疾病的一种潜在强大工具，从而导致其沉默。然而，为了使 siRNA 和 miRNA 分子有效地递送到感兴趣的器官/组织中，获得有效的体内递送，必须克服许多理化和生物学屏障。本综述讨论了与使用 siRNA 和 miRNA 相关的挑战，并描述了用于克服这些障碍的非病毒递药策略。更具体地说，重点介绍了那些已进展到用于局部和全身 siRNA 和 miRNA 递药的临床试验的技术。
