Wu B, Li YF, Morcos PA, Doran TJ, Lu PJ and Lu QL. Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice. Mol. Ther. advance online publication, March 10, 2009

http://www.nature.com/mt/journal/vaop/ncurrent/abs/mt200938a.html

Dystrophin exon 23 carries a nonsense mutation in the mdx mouse. Using a Morpholino oligo targeting an exon 23 splice junction can cause the splice machinery to skip exon 23, allowing translation of functional dystrophin with an internal deletion; however, unmodified Morpholinos enter cells poorly and large doses must be used to achieve splice modification in the mouse tissues. Coupling a Morpholino to a guanidinium dendrimer moiety makes a Vivo-Morpholino. When injected into the blood the Vivo-Morpholino circulates through the organism and enter cells, altering splicing and producing functional dystrophin.