Chemically modified mRNAs are extensively studied with a view toward their clinical application. In particular, long noncoding RNAs (lncRNAs) containing SINE elements, which enhance the translation of their target mRNAs (i.e., SINEUPs), have potential as RNA therapies for various diseases, such as haploinsufficiencies. To establish a SINEUP‐based system for efficient protein expression, we directly transfected chemically modified in vitro transcribed (mIVT) SINEUP RNAs to examine their effects on target mRNA translation. mIVT SINEUP RNAs enhanced translation of EGFP mRNA and endogenous target Sox9 mRNA in both cultured cells and a cell‐free translation system. Our findings reveal the functional role of RNA modifications in SINEUPs and suggest several broad clinical applications of such an RNA regulatory system.

中文翻译：

---

具有化学修饰的体外合成的 lncRNAs (SINEUPs) 增强了目标 mRNA 的翻译

为了它们的临床应用，对化学修饰的 mRNA 进行了广泛的研究。特别是，含有 SINE 元件的长链非编码 RNA (lncRNAs)，可增强其目标 mRNA（即 SINEUPs）的翻译，具有作为各种疾病（如单倍剂量不足）的 RNA 疗法的潜力。为了建立基于 SINEUP 的高效蛋白质表达系统，我们直接转染化学修饰的体外转录 (mIVT) SINEUP RNA，以检查它们对靶标 mRNA 翻译的影响。mIVT SINEUP RNA 增强了 EGFP mRNA 和内源性靶标 Sox9 mRNA 在培养细胞和无细胞翻译系统中的翻译。我们的研究结果揭示了 RNA 修饰在 SINEUP 中的功能作用，并提出了这种 RNA 调节系统的几种广泛临床应用。