Peptide nucleic acids: On the road to new gene therapeutic drugs

Abstract

Peptide nucleic acid (PNA) is a DNA mimic based on a pseudopeptide (polyamide) backbone. PNA oligomers bind strongly and with high sequence specificity to complementary targets in RNA (or DNA), and they show very high biological stability. Furthermore, studies in cell free systems have demonstrated potent antisense (inhibition of translation) and antigene (inhibition of transcription) activity of PNA. Recently, several studies reporting methods for cellular delivery of PNA as well as antisense effects of PNA in cells ex vivo and in rats have appeared. The potential of developing PNA derived gene therapeutic drugs is discussed.