Nonviral vector-mediated RNA interference: Its gene silencing characteristics and important factors to achieve RNAi-based gene therapy

作者：Takahashi Yuki; Nishikawa Makiya; Takakura Yoshinobu^*

来源：Advanced Drug Delivery Reviews, 2009, 61(9): 760-766.

DOI：10.1016/j.addr.2009.04.006

• Short hairpin RNA
• In vivo delivery
• Duration
• Side effect
• Hydrodynamic delivery

RNA interference (RNAi) is a potent and specific gene silencing event in which small interfering RNA (siRNA) degrades target mRNA. Therefore, RNAi is of potential use as a therapeutic approach for the treatment of a variety of diseases in which aberrant expression of mRNA causes a problem. RNAi can be achieved by delivering siRNA or vectors that transcribe siRNA or short-hairpin RNA (shRNA). The aim of this review is to examine the potential of nonviral vector-mediated RNAi technology in treating diseases. The characteristics of plasmid DNA expressing shRNA were compared with those of siRNA, focusing on the duration of gene silencing, delivery to target cells and target specificity. Recent progresses in prolonging the RNAi effect, improving the delivery to target cells and increasing the specificity of RNAi in vivo are also reviewed.

• 出版日期2009-7-25