Morpholinos are short antisense oligos that can bind to and thereby block an exon’s expression, resulting in an imperfect but still functional protein.  Researchers have been exploring their utility in model organisms since James Summerton and Dwight Weller first characterized phosphorodiamidate morpholino oligos about a decade ago.  Now, a group at Children’s National Medical Center in Washington, DC with collaborators in North Carolina and Japan have used this approach, delivered by an injection into the blood stream, to treat Duchenne Muscular Dystrophy in the dog, laying the foundation for human trials.          MORE