Mendel-Brno 2000

category image Volume: 17
Issue Number 6, Part 2
June 2000

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.
Nielsen, P.E. (1999) Applications of peptide nucleic acids. Current Opinion in Biotechnology 10, 71-75.
Lohse, J., Dahl, O.& Nielsen, P.E.(1999) Double Duplex Invasion by Peptide Nucleic Acid: A general Principle for Sequnce Specific Targeting of Double Stranded DNA. Proc. Natl. Acad. Sci. USA 96, 11804-11808.
Ljungstrom, T., Knudsen, H. & Nielsen, P.E. (1999) Cellular Uptake of Adamantyl Conjugated Peptide Nucleic Acids. Bioconjug Chem. 10, 965-972.

P. E. Nielsen

University of Copenhagen, IMBG, Laboratory B,
The Panum Institute,
Blegdamsvej 3, DK-2200 Copenhagen N, Denmark