Exon 53 of dystrophin pre-mRNA

Exon 53 of dystrophin pre-mRNA is one of 79 protein-coding exons in the DMD gene pre-mRNA that, when correctly spliced, enables the production of full-length dystrophin—a structural protein essential for muscle fiber stability. In Duchenne muscular dystrophy, certain exon deletions disrupt the reading frame, causing the absence of functional dystrophin. Drugs such as viltolarsen and golodirsen are designed to bind exon 53 in the pre-mRNA and induce its skipping during splicing, restoring the mRNA's reading frame and enabling the synthesis of a truncated but functional dystrophin protein, thereby providing a molecularly guided therapy for a subset of DMD patients

Other names

Exon 53 of the DMD geneDMD exon 53

Antisense oligonucleotide-mediated exon skipping: Synthetic oligonucleotides bind to exon 53 of dystrophin pre-mRNA, hiding it from the spliceosome and causing its exclusion from the mature mRNA, restoring the transcript’s reading frame and enabling production of a truncated but partially functional dystrophin protein