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 an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …

Whole-body systemic gene therapy is likely the most effective way to reduce greatly the
disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle …

Background Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder with
monogenic mutations setting the stage for successful gene therapy treatment. We have …

Dystrophin deficiency leads to lethal dilated Duchenne cardiomyopathy. A promising
therapy is to deliver a highly abbreviated microdystrophin gene to the heart using adeno …

Dual adeno-associated virus (AAV) technology was developed in 2000 to double the
packaging capacity of the AAV vector. The proof of principle has been demonstrated in …

Abstract Adeno-associated virus (AAV)-mediated microdystrophin gene therapy holds great
promise for treating Duchenne muscular dystrophy (DMD). Previous studies have revealed …

Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease
caused by mutations in the dystrophin gene. Gene therapy using highly functional …

Duchenne muscular dystrophy (DMD) is a lethal disorder of skeletal muscle caused by
mutations in the dystrophin gene. Adeno-associated virus (AAV) vector-mediated gene …