title: 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.
authors.affiliation: Center for Biomolecular Recognition, Department of Medical Biochemistry & Genetics, Panum Institute, Copenhagen N, Denmark. pen@imbg.ku.dk
authors.forename: P E
authors.initials: PE
authors.lastname: Nielsen
mesh_terms: D000595:Amino Acid Sequence; D000818:Animals; D016375:Antisense Elements (Genetics); D001483:Base Sequence; D004247:DNA / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D018716:Molecular Mimicry; D008969:Molecular Sequence Data; D020135:Peptide Nucleic Acids / Q000235:genetics / Q000494:pharmacology / Q000627:therapeutic use*
chemical_list: D016375:Antisense Elements (Genetics); D020135:Peptide Nucleic Acids; D004247:DNA