title: Sequence-selective targeting of duplex DNA by peptide nucleic acids.
abstract: Sequence-selective gene targeting constitutes an attractive drug-discovery approach for genetic therapy, with the aim of reducing or enhancing the activity of specific genes at the transcriptional level, or as part of a methodology for targeted gene repair. The pseudopeptide DNA mimic peptide nucleic acid (PNA) can recognize duplex DNA with high sequence specificity and affinity in triplex, duplex and double-duplex invasive modes or non-invasive triplex modes. Novel PNA modification has improved the affinity for DNA recognition via duplex invasion, double-duplex invasion and triplex recognition considerably. Such modifications have also resulted in new approaches to targeted gene repair and sequence-selective double-strand cleavage of genomic DNA.
authors.affiliation: University of Copenhagen, The Panum Institute, Department of Cellular and Molecular Medicine, Blegdamsvej 3c, DK-2200, Copenhagen, Denmark. ptrn@sund.ku.dk
authors.forename: Peter E
authors.initials: PE
authors.lastname: Nielsen
mesh_terms: D001483:Base Sequence; D004247:DNA / Q000737:chemistry / Q000235:genetics / Q000378:metabolism; D015195:Drug Design; D018390:Gene Targeting / Q000379:methods*; D006801:Humans; D008969:Molecular Sequence Data; D015394:Molecular Structure; D009690:Nucleic Acid Conformation; D020135:Peptide Nucleic Acids / Q000737:chemistry / Q000235:genetics / Q000378:metabolism*
chemical_list: D020135:Peptide Nucleic Acids; D004247:DNA