Peptide Nucleic Acid (PNA): Towards Gene Therapeutic Drugs
The pseudopeptide DNA mimic PNA (peptide nucleic acid) has many of the properties desired of a gene targeting reagent. It binds strongly and with high sequence specificity to complementary RNA and to homopurine and AT-rich targets in double stranded DNA, and can thereby specifically and efficiently inhibit translation and transcription, respectively. Furthermore, PNA has high biostability and it is easy to chemically synthesize and modify. In the process of evaluating the prospects of developing PNA derived gene therapeutic drugs, recent results relating to targeting of double stranded DNA, antisense gene regulation in bacteria and eukaryotic cells as well as improved methods for cellular uptake of PNA will be presented.
Good, L. & Nielsen, P.E. (1998) Antisense inhibition of gene expression in bacteria by PNA targeted to mRNA. Nature Biotechnology 16, 355-358.
P. E. Nielsen
University of Copenhagen, IMBG, Laboratory B,