Preparation and biofunctional evaluation of PNA-PEG conjugates that can be applied to the genetic diseases caused by single nucleotide mutations.

我们合成了PNA-聚乙二醇偶联物，并试图利用细胞外蛋白表达系统来调节基因的表达。结果表明，PNA-聚乙二醇偶联物有效地抑制了蛋白质的表达，并在体内识别了与Homo PNA寡聚体相比的一个碱基的差异。这是由于其在30℃下的热稳定性所致，这种类型的PNA-聚乙二醇偶联物以大吞饮的形式转运，但不能释放到胞浆中。在多肽信号(Q7-G-HSP)的作用下，PNA-聚乙二醇偶联物从巨噬细胞释放到胞浆中，但其细胞毒作用不容忽视。

We synthesized the PNA-PEG conjugate and tried to regulate a gene expression using the cell free protein expression systems. As a result, the PNA-PEG conjugate inhibited the protein expression effectively and recognized the difference of the one base in vivo, compared with homo PNA oligomer. This was caused by the thermal stability at 30 degree, and this type of PNA-PEG conjugate was transported with macropinocytosis, but couldn't release to cytosol. with peptide signal (Q7-G-HSP), PNA-PEG conjugate was released to cytosol from mcropinosome, but it showed cytotoxicity that can not be ignored.