Antisense oligonucleotides (ASOs) represent a new and highly promising class of drugs for
personalized medicine. In the last decade, major chemical developments and improvements …

The introduction of genetics revolutionized the field of neurodegenerative and
neuromuscular diseases and has provided considerable insight into the underlying …

Current genome-editing systems generally rely on inducing DNA double-strand breaks
(DSBs). This may limit their utility in clinical therapies, as unwanted mutations caused by …

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

Adequate skeletal muscle plasticity is an essential element for our well-being, and
compromised muscle function can drastically affect quality of life, morbidity, and mortality …

Regeneration of skeletal muscles is limited in cases of volumetric muscle loss and muscle
degenerative diseases. Therefore, there is a critical need for developing strategies that …

Background: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations,
resulting in absence of functional dystrophin protein. Viltolarsen, an exon 53 skipping …

Abstract Duchenne Muscular Dystrophy (DMD) is a lethal disease caused by mutations in
dystrophin encoding gene, causing progressive degeneration of cardiac, respiratory, and …

Limb–girdle muscular dystrophies (LGMDs) are caused by mutations in multiple genes. This
review article presents 39 genes associated with LGMDs. Some forms are inherited in a …

Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive
pediatric muscle disorder and genetically inherited as an X-linked disease that caused by …