Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease characterized by
progressive muscle wasting due to modifications in the DMD gene (exon deletions …

Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting
complications such as loss of skeletal muscle function as well as respiratory and cardiac …

Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are
currently in clinical trials, but have not been thoroughly investigated for their efficacy on …

For 15 years, gene therapy has been viewed as a beacon of hope for inherited retinal
diseases. Many preclinical investigations have centered around vectors with maximal gene …

Rationale: Absence of dystrophin in Duchenne muscular dystrophy (DMD) results in the
degeneration of skeletal and cardiac muscles. Owing to advances in respiratory …

Troponin I (TnI) is a key regulator of cardiac contraction and relaxation with TnI Ser-23/24
phosphorylation serving as a myofilament mechanism to modulate cardiac function. Basal …

Adeno-associated virus (AAV)-mediated systemic micro-dystrophin (μDys) therapy is
currently in clinical trials. The hope is to permanently improve the life quality of Duchenne …

Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease caused by
mutations to the dystrophin gene, resulting in deficiency of dystrophin protein, loss of …

Chronic cardiac muscle inflammation and fibrosis are key features of Duchenne Muscular
Dystrophy (DMD). Around 90% of 18-year-old patients already show signs of DMD-related …

This review summarizes the pathogenic mechanisms that underpin the monogenic
epilepsies and discusses the potential of novel precision therapeutics to treat these …