Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and generally survive into their twenties. There is currently no effective treatment except palliative care, although personalized treatments such as exon skipping, stop codon read-through, and viral-based gene therapies are making progress. Patients...

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