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-PEG偶联物，并尝试使用细胞游离蛋白表达系统来调节基因表达。结果表明，与同源PNA低聚物相比，PNA- peg偶联物有效抑制了蛋白的表达，并在体内识别了一个碱基的差异。这是由于30度的热稳定性造成的，这种类型的PNA-PEG偶联物在巨噬细胞作用下运输，但不能释放到细胞质中。通过肽信号（Q7-G-HSP）， PNA-PEG偶联物从微酶体释放到胞质中，但其细胞毒性不容忽视。

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.