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Translational Medicine: Open Access(TMOA)

ISSN: 2993-5407 | DOI: 10.33140

Molecular Therapies,

Molecular therapies for neuromuscular disorders are rapidly expanding and hold promise for devastating diseases with few options for treatment. Diseases targeted in this review include Duchenne, Becker, and the limb girdle muscular dystrophies. Strategies to repair defective genes involve exon skipping using antisense oligonucleotides or mutation suppression requiring small molecules that permit dose-dependent readthrough of stop codons.