Duchenne muscular dystrophy (DMD) is an X-linked lethal muscle disease caused by
dystrophin deficiency. Gene therapy has significantly improved the outcome of dystrophin …

A milestone of molecular medicine is the identification of dystrophin gene mutation as the
cause of Duchenne muscular dystrophy (DMD). Over the last 2 decades, major advances in …

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 …

Duchenne muscular dystrophy (DMD) is one of the most common lethal, hereditary diseases
of childhood. Since the identification of the genetic basis of this disorder, there has been the …

Duchenne muscular dystrophy (DMD) is a devastating X‐linked muscle disorder
characterized by muscle wasting which is caused by mutations in the DMD gene. The DMD …

Experiments in dystrophin gene transgenic mice have supported the concept of treating
Duchenne muscular dystrophy (DMD) by demonstrating that regional expression of …

Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, and …

Introduction Duchenne muscular dystrophy (DMD) is one of the most severe and devastating
neuromuscular hereditary diseases with a male newborn incidence of 20 000 cases each …

Gene therapy for Duchenne muscular dystrophy : Current Opinion in Neurology Gene therapy
for Duchenne muscular dystrophy : Current Opinion in Neurology Log in or Register Subscribe …

Therapies for Duchenne muscular dystrophy (DMD) must first be tested in animal models to
determine proof-of-concept, efficacy, and importantly, safety. The murine and canine models …