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 for sequence-specific interference with pre-mRNA splicing, thus offering various applications in fundamental research and in therapeutics. The chemical synthesis of these CPP–ON conjugates will be described as well as easy-to-implement assays to monitor cellular uptake, endosome leakage, and efficiency of splicing redirection.
CITATION STYLE
Abes, R., Arzumanov, A. A., Saleh, A. F., Hassane, F. S., Gait, M. J., & Lebleu, B. (2011). Splice Redirection as a Convenient Assay to Monitor CPP–ON Efficiency and Mechanism. In Methods in Molecular Biology (Vol. 683, pp. 307–320). Humana Press Inc. https://doi.org/10.1007/978-1-60761-919-2_22
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