U1sn RNA是一种富含尿苷酸的具有酶活性的小分子量 RNA,其主要特点是剪接核内非均一性 RNA成为成熟 RNA;只存在于真核细胞核质中 ,具有在核内定位的特性。有人利用这些特性对 U1sn RNA进行人工点突变来纠正某些基因缺陷病 ,或是构建 U1sn RNA-核酶及 U1sn RNA-反义核酸的嵌合体来进行基因治疗研究并获得了一定成绩 U1sn RNA is a small, uridine-rich, enzymatically-active small RNA that splices in-core heterogeneous RNA into mature RNA; exists only in eukaryotic cytoplasm and possesses nuclear localization properties . Some people use these features to artificially point mutation U1sn RNA to correct some genetic defects, or to construct U1sn RNA-ribozyme and U1sn RNA-antisense nucleic acid chimeras for gene therapy research and achieved some results