Targeting Noncoding RNAs in Disease: Challenges and Opportunities

Noncoding RNAs make up the majority of transcribed RNA and have a wide range of functions in cellular and developmental processes. Consequently, they are also implicated in the development and pathophysiology of many diseases and represent potential targets for therapeutic intervention.

Scientists found that effective in vivo inhibition (or silencing) of microRNA, one class of noncoding RNA, has enabled to make groundbreaking discoveries about the contribution of these short regulating RNAs to some of the major human diseases, such as cancer, heart disease, and diabetes. Long noncoding RNAs are less well studied, but have recently emerged as another novel class of therapeutic targets in a variety of diseases. Both long and short noncoding RNAs represent new avenues of investigation for drug discovery with several advantages over traditional protein-based targets; however, they come with their own unique set of challenges.

Source: For more details about these findings, please view the Science webinar (Corey et al. Targeting Noncoding RNAs in Disease: Challenges and Opportunities. Science 30 August 2013: Vol. 341 no. 6149 p. 1021)