Summary
Over the past several years, advances in RNA sequencing have led to an increased appreciation of the prevalence and function of noncoding RNAs, including long noncoding RNAs (lncRNAs). These are typically expressed in a tissue-specific manner in healthy tissues and are often dysregulated in disease, making them potential biomarkers and therapeutic targets. On page 662 of this issue, Li et al. (1) reveal the biological importance of a lncRNA in an inherited metabolic disorder called phenylketonuria (PKU) and demonstrate in mice that a molecule that mimics the functional region of this lncRNA is a promising therapeutic. This discovery suggests that short lncRNA fragments could overcome some of the challenges faced by other RNA therapeutic modalities.
