Peptide-based delivery of steric-block PNA oligonucleotides

Autor: Abes, Saïd, Ivanova, Gabriela D, Abes, Rachida, Arzumanov, Andrey A, Williams, Donna, Owen, David, Lebleu, Bernard, Gait, Michael J
Přispěvatelé: Dynamique des interactions membranaires normales et pathologiques ( DIMNP ), Université Montpellier 1 ( UM1 ) -Université Montpellier 2 - Sciences et Techniques ( UM2 ) -Université de Montpellier ( UM ) -Centre National de la Recherche Scientifique ( CNRS ), Engelhardt Institute of Molecular Biology ( ISTC ), Russian Academy of Sciences [Moscow] ( RAS ), Centre for Ecology and Hydrology [Bangor] ( CEH ), Natural Environment Research Council ( NERC ), National Hansen's Disease Program, Lousiana State University, Dynamique des interactions membranaires normales et pathologiques (DIMNP), Centre National de la Recherche Scientifique (CNRS)-Université de Montpellier (UM)-Université Montpellier 2 - Sciences et Techniques (UM2)-Université Montpellier 1 (UM1), Engelhardt Institute of Molecular Biology (ISTC), Russian Academy of Sciences [Moscow] (RAS)
Rok vydání: 2008
Předmět:
Zdroj: Methods in Molecular Biology (Clifton then Totova)
Methods in Molecular Biology (Clifton then Totova), Humana Press (Springer Imprint), 2009, 480, pp.85-99. 〈10.1007/978-1-59745-429-2_6〉
Methods in Molecular Biology
Methods in Molecular Biology, Humana Press/Springer Imprint, 2009, 480, pp.85-99. ⟨10.1007/978-1-59745-429-2_6⟩
ISSN: 1064-3745
1940-6029
Popis: International audience; 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.
Databáze: OpenAIRE