Peptide nucleic acid (PNA) is a DNA mimic based on a pseudopeptide (polyami
de) backbone. PNA oligomers bind strongly and with high sequence specificit
y to complementary targets in RNA (or DNA), and they show very high biologi
cal stability. Furthermore, studies in cell free systems have demonstrated
potent antisense (inhibition of translation) and antigene (inhibition of tr
anscription) activity of PNA. Recently several studies reporting methods fo
r 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 gen
e therapeutic drugs is discussed.