Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

Background Major efforts have been made in the last decade to develop and improve
therapies for proximal spinal muscular atrophy (SMA). The introduction of …

Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is
characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete …

To maintain and restore skeletal muscle mass and function is essential for healthy aging. We
have found that myonectin acts as a cardioprotective myokine. Here, we investigate the …

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder with
a prevalence of approximately 1 in 3500–5000 males. DMD manifests as childhood-onset …

Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder characterized by
progressive muscle wasting that has currently no cure. Exon-skipping strategy represents …

Dystrophin proteomic regulation in muscular dystrophies (MDs) remains unclear. We report
that a long noncoding RNA (lncRNA), H19, associates with dystrophin and inhibits E3-ligase …

Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive
muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense …