title: Peptide-based delivery of steric-block PNA oligonucleotides.
abstract: Several strategies based on synthetic oligonucleotides (ON) have been proposed to control gene expression. As for most biomolecules, however, delivery has remained a major roadblock for in vivo applications. Conjugation of steric-block neutral DNA mimics such as peptide nucleic acids (PNA) or phosphorodiamidate morpholino oligonucleotides (PMO) to cell penetrating peptides (CPP) has recently been proposed as a new delivery strategy. It is particularly suitable to interfere sequence-specifically with pre-mRNA splicing thus offering various applications in fundamental research and in therapeutics. The chemical synthesis of these CPP conjugates as well as methodologies to monitor their cellular uptake and their efficiency in a reliable and easy to implement assay of splicing correction will be described.
authors.affiliation: UMR 5235 CNRS, Université, Montpellier 2, Place Eugene Bataillon, 34095 Montpellier cedex 5, France.
authors.forename: Saïd
authors.initials: S
authors.lastname: Abes
authors.forename: Gabriela D
authors.initials: GD
authors.lastname: Ivanova
authors.forename: Rachida
authors.initials: R
authors.lastname: Abes
authors.forename: Andrey A
authors.initials: AA
authors.lastname: Arzumanov
authors.forename: Donna
authors.initials: D
authors.lastname: Williams
authors.forename: David
authors.initials: D
authors.lastname: Owen
authors.forename: Bernard
authors.initials: B
authors.lastname: Lebleu
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Gait
mesh_terms: D002469:Cell Separation; D005434:Flow Cytometry; D006367:HeLa Cells; D006801:Humans; D008856:Microscopy, Fluorescence; D009841:Oligonucleotides / Q000008:administration & dosage*; D020135:Peptide Nucleic Acids / Q000008:administration & dosage*; D010455:Peptides / Q000737:chemistry*; D020133:Reverse Transcriptase Polymerase Chain Reaction
chemical_list: D009841:Oligonucleotides; D020135:Peptide Nucleic Acids; D010455:Peptides