Neurological disorders are primarily diseases with sophisticated etiology that are always
refractory and recrudescent. The major obstruction to effective therapies for neurological …

Duchenne muscular dystrophy (DMD) is a debilitating genetic disorder that results in
progressive muscle degeneration and premature death. DMD is caused by mutations in the …

Duchenne muscular dystrophy (DMD) is a fatal X-linked disease caused by mutations in the
DMD gene, leading to complete absence of dystrophin and progressive degeneration of …

Duchenne muscular dystrophy (DMD), a severe hereditary disease affecting 1 in 3,500 boys,
mainly results from the deletion of exon (s), leading to a reading frameshift of the DMD gene …

Rationale: Autosomal-dominant mutations in ryanodine receptor type 2 (RYR2) are
responsible for≈ 60% of all catecholaminergic polymorphic ventricular tachycardia …

Precise gene manipulation by gene editing approaches facilitates the potential to cure
several debilitating genetic disorders. Gene modification stimulated by engineered …

Gene correction is a valuable strategy for treating inherited retinal degenerative diseases, a
major cause of irreversible blindness worldwide. Single gene defects cause the majority of …

Cardiovascular diseases are among the main causes of morbidity and mortality in Western
countries and considered as a leading public health issue. Therefore, there is a strong need …

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-
of-function mutations in the dystrophin gene and characterized by progressive muscle …

Gene therapy is based on the principle of the genetic manipulation of DNA or RNA for
treating and preventing human diseases. The clustered regularly interspaced short …