Destroying RNA as a therapeutic approach

Curr Med Chem. 2006;13(8):863-81. doi: 10.2174/092986706776361021.

Abstract

The ability to target RNA, mRNA and viral RNA in particular, for degradation is a powerful approach in molecular biology and pharmacology. Such approaches can be used in the study of gene function as in functional genomics, in the identification of disease-associated genes, and for the treatment of human diseases. This review provides a comprehensive up-to-date look at all the current available technologies used for the destruction of RNA, with a focus on their therapeutic potential. This includes approaches that utilize the activity of protein ribonucleases such as antisense oligonucleotide, small interfering RNA, RNase P-associated external guide sequence, onconase and bovine seminal RNase. Sequence-specific approaches that do not utilize activity of protein ribonucleases, such as ribozyme and DNazyme, are also reviewed and discussed. This review should provide a useful starting framework for researchers interested in using the RNA-destruction methodologies on the bench and in the clinic, and serves as a stimulus for further development of novel and more potent RNA degradation technologies. This is particularly critical, given the anticipation of discoveries of new cellular RNA degradation machineries and human diseases that are associated with dysfunctional RNA molecules.

Publication types

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

MeSH terms

  • Animals
  • Humans
  • Neoplasms / drug therapy
  • RNA / drug effects*
  • RNA, Catalytic / pharmacology
  • RNA, Messenger / drug effects
  • RNA, Small Interfering / antagonists & inhibitors
  • RNA, Viral / drug effects
  • Ribonucleases / pharmacology
  • Virus Diseases / drug therapy

Substances

  • RNA, Catalytic
  • RNA, Messenger
  • RNA, Small Interfering
  • RNA, Viral
  • RNA
  • Ribonucleases