Small RNA- and DNA-based gene therapy for the treatment of liver cirrhosis, where we are?

World J Gastroenterol. 2014 Oct 28;20(40):14696-705. doi: 10.3748/wjg.v20.i40.14696.

Abstract

Chronic liver diseases with different aetiologies rely on the chronic activation of liver injuries which result in a fibrogenesis progression to the end stage of cirrhosis and liver failure. Based on the underlying cellular and molecular mechanisms of a liver fibrosis, there has been proposed several kinds of approaches for the treatment of liver fibrosis. Recently, liver gene therapy has been developed as an alternative way to liver transplantation, which is the only effective therapy for chronic liver diseases. The activation of hepatic stellate cells, a subsequent release of inflammatory cytokines and an accumulation of extracellular matrix during the liver fibrogenesis are the major obstacles to the treatment of liver fibrosis. Several targeted strategies have been developed, such as antisense oligodeoxynucleotides, RNA interference and decoy oligodeoxynucleotides to overcome this barriers. With this report an overview will be provided of targeted strategies for the treatment of liver cirrhosis, and particularly, of the targeted gene therapy using short RNA and DNA segments.

Keywords: Antisense; Decoy; Gene therapy; Liver cirrhosis; Micro RNA; Oligodeoxynucleotides; Small interfering RNA.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Gene Expression Regulation
  • Genetic Therapy / methods*
  • Humans
  • Liver Cirrhosis / genetics
  • Liver Cirrhosis / metabolism
  • Liver Cirrhosis / therapy*
  • MicroRNAs / therapeutic use*
  • Oligonucleotides, Antisense / therapeutic use*
  • RNA Interference*
  • RNA, Small Interfering / therapeutic use*
  • Treatment Outcome

Substances

  • MicroRNAs
  • Oligonucleotides, Antisense
  • RNA, Small Interfering