Research progress on siRNA delivery with nonviral carriers

Yan Gao^*, Xin-Ling Liu^*, Xiao-Rong Li
Tianjin Medical University Eye Center, Tianjin, China
^*These two authors contributed equally to this work

Abstract: RNA interference is a powerful method for the knockdown of pathologically relevant genes. Small interfering RNAs (siRNAs) have been widely demonstrated as effective biomedical genetic-therapy applications for many diseases. Unfortunately, siRNA duplexes are not ideal drug-like molecules. Problems hindering their effective application fundamentally lie in their delivery, stability, and off-target effects. Delivery systems provide solutions to many of the challenges facing siRNA therapeutics. Due to some fatal disadvantages of viral vectors, nonviral carriers have been studied extensively. Aside from liposomes, nanoparticles and cationic polymer carriers have exhibited improved in vivo stability, better biocompatibility, and efficiency for gene silencing with less cellular toxicity. They may represent a promising strategy for siRNA-based therapies, especially as nanomaterials. The present review also summarizes other methods of siRNA delivery and the side effects of the nanoparticles.

Keywords: small interfering RNA, nonviral vector, gene therapy, delivery system, nanoparticles, biocompatibility