PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation

@inproceedings{Ezzat2011PepFect1A,
  title={PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation},
  author={Kariem Ezzat and Samir A El Andaloussi and Eman M. Zaghloul and Taavi Lehto and Staffan A Lindberg and Pedro M. D. Moreno and Joana R. Viola and Tarek Magdy and Rania Abdo and Peter Guterstam and Rannar Sillard and Suzan M Hammond and Matthew J. Wood and Andrey A. Arzumanov and Michael J. Gait and C I Edvard Smith and Mattias M H{\"a}llbrink and {\"U}lo Langel},
  booktitle={Nucleic acids research},
  year={2011}
}
Numerous human genetic diseases are caused by mutations that give rise to aberrant alternative splicing. Recently, several of these debilitating disorders have been shown to be amenable for splice-correcting oligonucleotides (SCOs) that modify splicing patterns and restore the phenotype in experimental models. However, translational approaches are required to transform SCOs into usable drug products. In this study, we present a new cell-penetrating peptide, PepFect14 (PF14), which efficiently… CONTINUE READING