Vivo-Morpholinos used in a mouse model of Duchenne muscular dystrophy:
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
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.
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Antisense delivery in adult mice
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