To treat a mouse model of Duchenne muscular dystrophy, molecules of the exosome-binding peptide CP05 are linked either to the muscle-targeting peptide M12 or an oligomer (PMO) that corrects a splicing error in the gene for dystrophin. These two conjugate peptides paint the surface of exosomes from cultured mouse cells. When injected into the mice, the exosomes home to muscle, where they deliver the oligomer, boost functional dystrophin levels, and improve muscle function.

© george retseck


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