Peptide-based delivery of steric-block PNA oligonucleotides.
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.
Abes
Saïd
S
UMR 5235 CNRS, Université, Montpellier 2, Place Eugene Bataillon, 34095 Montpellier cedex 5, France.
Ivanova
Gabriela D
GD
Abes
Rachida
R
Arzumanov
Andrey A
AA
Williams
Donna
D
Owen
David
D
Lebleu
Bernard
B
Gait
Michael J
MJ
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
D009841:Oligonucleotides; D020135:Peptide Nucleic Acids; D010455:Peptides