The last few decades have seen an explosion in identification of genes that cause
monogenetic neurological diseases, as well as advances in gene-targeting therapeutics …

Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD
reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an …

Ribitol-phosphate modification is crucial for the functional maturation of α-dystroglycan. Its
dysfunction is associated with muscular dystrophy, cardiomyopathy, and central nervous …

Cardiovascular diseases are the most common cause of worldwide morbidity and mortality,
highlighting the necessity for advanced therapeutic strategies. Ca2+/calmodulin-dependent …

The essential product of the Duchenne muscular dystrophy (DMD) gene is dystrophin, a rod-
like protein that protects striated myocytes from contraction-induced injury,. Dystrophin …

Creatine deficiency syndromes (CDS) are inherited metabolic disorders caused by
mutations in GATM, GAMT and SLC6A8 and mainly affect central nervous system (CNS) …

During the last decade, multiple clinical trials for Duchenne muscular dystrophy (DMD) have
focused on the induction of dystrophin expression using different strategies. Many of these …

Duchenne muscular dystrophy, like other muscular dystrophies, is a progressive disorder
hallmarked by muscle degeneration, inflammation, and fibrosis. Latent transforming growth …

The emergence of CRISPR-Cas9 gene-editing technologies and genome-wide CRISPR-
Cas9 libraries enables efficient unbiased genetic screening that can accelerate the process …

Treatment of monogenetic disorders using vectors based on adeno-associated virus (AAV)
is an area of intense interest. AAV is non-pathogenic human virus, and preexisting capsid …