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 a progressive neuromuscular disease caused by
mutations to the dystrophin gene, resulting in deficiency of dystrophin protein, loss of …

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

Adeno-associated virus–mediated (AAV-mediated) CRISPR editing is a revolutionary
approach for treating inherited diseases. Sustained, often life-long mutation correction is …

Duchenne muscular dystrophy (DMD) represents one of the most devastating types of
muscular dystrophies which affect boys already at early childhood. Despite the fact that the …

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

Loss of dystrophin leads to Duchenne muscular dystrophy (DMD). A pathogenic feature of
DMD is the significant elevation of cytosolic calcium. Supraphysiological calcium triggers …

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive condition caused
primarily by out-of-frame mutations in the dystrophin gene. In males, DMD presents with …

Gene replacement for Duchenne muscular dystrophy (DMD) with micro-dystrophins has
entered clinical trials, but efficacy in preventing heart failure is unknown. Although most …

Aims The mdx mouse has proven to be useful in understanding the cardiomyopathy that
frequently occurs in muscular dystrophy patients. Here we employed a comprehensive array …