Science Webinar Series

Targeting Noncoding RNAs in Disease: Challenges and Opportunities

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Science  30 Aug 2013:
Vol. 341, Issue 6149, pp. 1021
DOI: 10.1126/science.341.6149.1021-c


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. microRNAs are one class of noncoding RNA that has been intensely studied. Effective inhibition (or silencing) of microRNAs in vivo has enabled scientists 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.