Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Micro-dystrophins are highly promising candidates for treating Duchenne muscular
dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust …

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy.
There is no effective treatment and patients typically die in approximately the third decade …

Duchenne muscular dystrophy (DMD) is a genetic muscle disorder caused by mutations in
the Dmd gene resulting in the loss of the protein dystrophin. Patients do not only experience …

Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is
caused by mutations to the DMD gene that encodes the dystrophin protein. Recent …

Aims The cardiomyopathy found in Duchenne muscular dystrophy (DMD) is responsible for
death due to heart failure in∼ 30% of patients and additionally contributes to many DMD …

Introduction: Muscle-directed gene therapy is rapidly gaining attention primarily because
muscle is an easily accessible target tissue and is also associated with various severe …

Dystrophin is a large protein serving as local scaffolding repetitively bridging cytoskeleton
and the outside of striated muscle cell. As such dystrophin is a critical brick primarily in …

Animal models for Duchenne muscular dystrophy (DMD) have species limitations related to
assessing function, immune response, and distribution of micro-or mini-dystrophins …