1 2 aim

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 […]