In the past decade, the development of two innovative technologies, namely, induced
pluripotent stem cells (iPSCs) and the CRISPR Cas9 system, has enabled researchers to …

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. Mutations of the DMD gene destabilize the dystrophin associated glycoprotein …

Duchenne muscular dystrophy is an X-linked monogenic disease caused by mutations in
the dystrophin gene (DMD) characterized by progressive muscle weakness, leading to loss …

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 genetic neuromuscular disease that primarily
affects young males. Patients with DMD are unable to produce dystrophin, a crucial protein …

Background Currently, the most promising therapies for Duchenne muscular dystrophy
(DMD) are exon skipping and stop codon read-through, two strategies aimed at restoring the …

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 …

Duchenne muscular dystrophy (DMD) affecting 1 in 3500–5000 live male newborns is the
frequently fatal genetic disease resulted from various mutations in DMD gene encoding …

Dystrophinopathies are allelic conditions caused by deletions, duplications and point-
mutations in the DMD gene, located on the X chromosome (Xp21. 2). Mutations that …

The first trial, using intramuscular drisapersen, increased sarcolemmal dystrophin in 64–
97% of examined myofibres; subsequent phase II/III clinical trials using systemic drisapersen …