Therapeutic Applications of RNA Interference


During recent decades there have been remarkable advances in biology, in which one of the most important discoveries is ribonucleic acid interference (RNAi). RNAi is a specific post‐transcriptional regulatory pathway that can result in silencing gene functions. RNAi products include small interfering ribonucleic acid (siRNA) and also short hairpin ribonucleic acid (shRNA), microRNA (miRNA) and Piwi‐interacting ribonucleic acid (piRNA). They combine with homologous fragments of the mRNA (messenger ribonucleic acid) and cause its degradation. It results in inhibition of protein synthesis, or in mutation in the gene encoding it. Efforts have been taken to translate this new discovery into clinical applications for disease treatment. The basic and clinical research has been focused on the following targets: cancer, cardiovascular diseases, neurodegenerative diseases, virus‐related diseases as well as skin disorder and ocular disorders.

Key Concepts

  • RNA interference (RNAi) is a specific post‐transcriptional gene‐silencing mediated by small RNAs.
  • RNA‐induced silencing complex (RISC) is important for RNAi.
  • RNAi has become a potential treatment for human diseases.
  • RNAi can inhibit tumour cell growth, angiogenesis, metastasis and chemoresistance.
  • RNAi suppresses disease‐linked genes and has a potential for the treatment of cardiovascular neurodegenerative and virus‐related diseases.

Keywords: RNA interference; gene‐silencing; therapeutics; siRNA; miRNA


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Further Reading

Bora RS, Gupta D, Mukkur TK, et al. (2012b) RNA interference therapeutics for cancer: challenges and opportunities (review). Molecular Medicine Reports 6: 9–15.

Borna H, Imani S, Iman M, et al. (2015b) Therapeutic face of RNAi: in vivo challenges. Expert Opinion on Biological Therapy 15: 269–285.

Deng Y, Wang CC, Choy KW, et al. (2014) Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies. Gene 538: 217–227.

Kubowicz P, Zelaszczyk D and Pekala E (2013) RNAi in clinical studies. Current Medicinal Chemistry 20: 1801–1816.

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Deng, Yan, Chen, Jiao, Zhao, Yi, and Tang, Tao(Feb 2016) Therapeutic Applications of RNA Interference. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1002/9780470015902.a0026463]