Duchenne muscular dystrophy (DMD) is a severe, §progressive muscle wasting disease for which there §is currently no cure. The disease is caused by §mutations in the DMD gene that completely abolish §the function of the protein dystrophin. §This thesis focuses on the development of a §promising new therapy, so called antisense-mediated §exon skipping, that aims to restore the disrupted §dystrophin reading frame to allow generation of §internally deleted, but partly functional dystrophin §proteins, as found in the less severe Becker §muscular dystrophy.§The thesis describes the DMD...
Duchenne muscular dystrophy (DMD) is a severe, §progressive muscle wasting disease for which there §is currently no cure. The disease is caused by Â...