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.

在过去几年中，RNA 测序的进步导致人们对非编码 RNA 的流行和功能的认识增加，包括长非编码 RNA (lncRNA)。这些通常在健康组织中以组织特异性方式表达，并且在疾病中经常失调，使它们成为潜在的生物标志物和治疗靶点。在本期第 662 页上，Li等人。( 1 ) 揭示了 lncRNA 在称为苯丙酮尿症 (PKU) 的遗传性代谢紊乱中的生物学重要性，并在小鼠身上证明了模拟这种 lncRNA 功能区域的分子是一种很有前途的治疗方法。这一发现表明，短的 lncRNA 片段可以克服其他 RNA 治疗方式面临的一些挑战。