Local dystrophin restoration with antisense oligonucleotide PRO051

van Deutekom JC, Janson AA, Ginjaar IB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. The New England journal of medicine 2007;357:2677-86   Abstract BACKGROUND: Duchenne’s muscular dystrophy is associated with severe, progressive muscle weakness and typically leads to death between the ages of 20 and 35 years. By inducing specific exon skipping during […]