Although genetic and cell-mediated approaches are currently showing genuine promise in
preclinical and clinical trials, there remains considerable interest in the development of …

Duchenne muscular dystrophy (DMD) is caused by mutations in the gene that encodes the
427‐kDa cytoskeletal protein dystrophin. Increased knowledge of the function of dystrophin …

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 …

Since the identification of dystrophin as the protein product of the Duchenne and Becker
muscular dystrophy locus, many different mutations, encompassing the entire spectrum of …

Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …

Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Dystrophinopathies are diseases caused by mutations in the Duchenne Muscular Dystrophy
gene (DMD) encoding the dystrophin protein. Depending on the type of mutation, patients …

Duchenne muscular dystrophy is the most severe childhood form of muscular dystrophy
caused by mutations in the gene responsible for dystrophin production. There is no cure …

Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there
is currently no effective treatment except palliative therapy. There are several promising …

Since the discovery of the gene for Duchenne muscular dystrophy more than 20 years ago,
scientists have worked to apply molecular principles for restoration of the dystrophin protein …