Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Nonsense mutations promote premature translational termination and cause anywhere from
5–70% of the individual cases of most inherited diseases. Studies on nonsense-mediated …

Nonsense suppression therapy encompasses approaches aimed at suppressing translation
termination at in-frame premature termination codons (PTCs, also known as nonsense …

Termination of protein synthesis is not 100% efficient. A number of natural mechanisms that
suppress translation termination exist. One of them is STOP codon readthrough, the process …

Premature termination codons (PTCs) in the coding regions of mRNA lead to the incorrect
termination of translation and generation of non-functional, truncated proteins. Translational …

Nonsense-mediated decay (NMD) eliminates mRNAs containing premature termination
codons and thus helps limit the synthesis of abnormal proteins. New results uncover a …

Lafora disease is a severe, autosomal recessive, progressive myoclonus epilepsy. The
disease usually manifests in previously healthy adolescents, and death commonly occurs …

Highlights•Numerous pathophysiological features of DMD provide different therapeutic
avenues.•Pharmacolougical therapies target all muscles and are applicable to all DMD …

Objective The objective of this study was to establish the feasibility of long‐term gentamicin
dosing to achieve stop codon readthrough and produce full‐length dystrophin. Mutation …