Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Background Pharmacological corticosteroid therapy is the standard of care in Duchenne
Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression …

Background: Loss of dystrophin protein causes Duchenne muscular dystrophy (DMD),
characterized by progressive degeneration of cardiac and skeletal muscles, and mortality in …

Duchenne muscular dystrophy (DMD), a severe hereditary disease affecting 1 in 3,500 boys,
mainly results from the deletion of exon (s), leading to a reading frameshift of the DMD gene …

The CRISPR/Cas9 system is a great revolution in biology. This technology allows the
modification of genes in vitro and in vivo in a wide variety of living organisms. In most …

In-frame exon deletions of the Duchenne muscular dystrophy (DMD) gene produce
internally truncated proteins that typically lead to Becker muscular dystrophy (BMD), a milder …

Dystrophinopathies are X-linked diseases, including Duchenne muscular dystrophy and
Becker muscular dystrophy, due to DMD gene variants. In recent years, the application of …

Mutations disrupting the reading frame of the~ 2.4 Mb dystrophin-encoding DMD gene
cause a fatal X-linked muscle-wasting disorder called Duchenne muscular dystrophy (DMD) …

Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disorder generally caused by
out-of-frame mutations in the DMD gene. In contrast, in-frame mutations usually give rise to …

Exon skipping therapy has recently received attention for its ability to convert the phenotype
of lethal Duchenne muscular dystrophy (DMD) to a more benign form, Becker muscular …