A cationic morpholino antisense oligomer conjugate: synthesis, cellular uptake and inhibition of Gli1 in the hedgehog signalling pathway

A guanidinium-linked morpholino pentamer is synthesized and evaluated for cellular penetration properties. A regular morpholino and the pentamer conjugate can exhibit antisense effects when designed against Gli1-transcription factors of the Hh signalling pathway, which is a potential target for cancer therapy. Evidence was found from luciferase assays and western blot analysis.