Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy
Genetic researchers at Children’s National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of “multiple exon-skipping” to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse. Multiple exon-skipping employs multiple DNA-like molecules as a “DNA band-aids” to …