Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy

Abstract excerpt

Duchenne muscular dystrophy (DMD) causes profound and progressive muscle weakness and loss, resulting in early death. DMD is usually caused by frameshifting deletions in the gene DMD, which leads to absence of dystrophin protein. Dystrophin binds to F-actin and components of the dystrophin-associated glycoprotein complex and protects the sarcolemma from contraction-induced injury. Antisense...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Adipose Tissue and Metabolism
• Share to Exercise and Physiological Responses
• Share to Muscle Physiology and Disorders