RNA interference-based therapy and its delivery systems

Cancer Metastasis Rev. 2018 Mar;37(1):107-124. doi: 10.1007/s10555-017-9717-6.

Abstract

RNA interference (RNAi) is considered a highly specific approach for gene silencing and holds tremendous potential for treatment of various pathologic conditions such as cardiovascular diseases, viral infections, and cancer. Although gene silencing approaches such as RNAi are widely used in preclinical models, the clinical application of RNAi is challenging primarily because of the difficulty in achieving successful systemic delivery. Effective delivery systems are essential to enable the full therapeutic potential of RNAi. An ideal nanocarrier not only addresses the challenges of delivering naked siRNA/miRNA, including its chemically unstable features, extracellular and intracellular barriers, and innate immune stimulation, but also offers "smart" targeted delivery. Over the past decade, great efforts have been undertaken to develop RNAi delivery systems that overcome these obstacles. This review presents an update on current progress in the therapeutic application of RNAi with a focus on cancer therapy and strategies for optimizing delivery systems, such as lipid-based nanoparticles.

Keywords: Cancer therapy; Delivery systems; Nanoparticles; RNA interference.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell- and Tissue-Based Therapy
  • Drug Resistance, Neoplasm / genetics
  • Gene Transfer Techniques*
  • Genes, MDR
  • Genetic Therapy*
  • Humans
  • Molecular Targeted Therapy
  • Nanoparticles
  • Neoplasms / genetics*
  • Neoplasms / immunology
  • Neoplasms / therapy*
  • RNA Interference*
  • RNA, Small Interfering / genetics*
  • Stem Cells / metabolism

Substances

  • RNA, Small Interfering