golodirsen

Golodirsen (SRP-4053) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) class that restores the dystrophin gene reading frame in Duchenne muscular dystrophy by modulating pre-mRNA splicing to induce skipping of exon 53, thereby enabling partial dystrophin expression and restoring anti-myostatin protein levels, which supports its application as a molecular tool for studying exon-skipping strategies and disease-modifying approaches in DMD research.