title: Progress in developing PNA as a gene-targeted drug.
abstract: Peptide nucleic acid (PNA) is a DNA mimic in which the nucleobases are attached to a pseudopeptide backbone. This achiral, uncharged, and rather flexible peptide backbone permits more stable hybridization to DNA and RNA oligomers with uncompromised or even improved sequence selectivity. Additional advantages of PNA are stability against nucleases and proteases and convenient solid phase synthesis. At the RNA level, PNA can be targeted to mRNA to block protein synthesis in an antisense strategy. PNA can also be targeted to the RNA component of ribonucleoproteins (RNPs) to inhibit their enzymatic activities. At the DNA level, the unique ability of PNA to bind DNA by duplex invasion can be used to arrest transcription within a gene sequence or to provide an artificial open complex to promote transcription. This review focuses on recent progress toward the development of PNA as a sequence-targeted drug.
authors.affiliation: Department of Medical Biochemistry and Genetics, University of Copenhagen, Panum Institute, Copenhagen, Denmark.
authors.forename: L
authors.initials: L
authors.lastname: Good
authors.forename: P E
authors.initials: PE
authors.lastname: Nielsen
mesh_terms: D001483:Base Sequence; D004247:DNA / Q000737:chemistry*; D003851:Deoxyribonucleases; D004337:Drug Carriers; D010450:Endopeptidases; D009690:Nucleic Acid Conformation; D009696:Nucleic Acids; D010455:Peptides / Q000138:chemical synthesis / Q000494:pharmacology; D012313:RNA / Q000187:drug effects; D012270:Ribosomes / Q000187:drug effects / Q000378:metabolism; D014158:Transcription, Genetic / Q000187:drug effects
chemical_list: D004337:Drug Carriers; D009696:Nucleic Acids; D010455:Peptides; D012313:RNA; D004247:DNA; D003851:Deoxyribonucleases; D010450:Endopeptidases