PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation.
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...Expand abstract
- Publication status:
- Publisher copy:
- Copyright date: