Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review

Abstract excerpt

Duchenne muscular dystrophy is a devastating disease that leads to progressive muscle loss and premature death. While medical management focuses mostly on symptomatic treatment, decades of research have resulted in first therapeutics able to restore the affected reading frame of dystrophin transcripts or induce synthesis of a truncated dystrophin protein from a vector, with other strategies based on gene therapy...

Topics

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

• Share to Muscle Physiology and Disorders
• Share to Viral Infections and Immunology Research
• Share to Virus-based gene therapy research