Mutations in the Myotonic Dystrophy Protein Kinase gene (DMPK) cause an autosomal dominant inherited disease referred to as Myotonic Dystrophy. Myotonic Dystrophy affects more than 1 in 8,000 people worldwide. Myotonic dystrophy results in progressive muscle weakness, stiffness and wasting. Currently, here are no treatments for this disease. A team of researchers at Nationwide Children’s Hospital have designed short hairpin constructs of AAV-shRNA which can be used to silence DMPK mRNA and therefore potentially treat myotonic dystrophy.