# Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells

**Authors:** Naoki Suzuki

PMC · DOI: 10.1016/j.omtn.2025.102462 · 2025-02-04

## Full-text entities

- **Genes:** DYSF (dysferlin) [NCBI Gene 8291] {aka FER1L1, LGMD2B, LGMDR2, MMD1}
- **Diseases:** dysferlinopathy (MESH:C537995)
- **Chemicals:** Antisense oligonucleotide (MESH:D016376)
- **Species:** Homo sapiens (human, species) [taxon 9606]

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Source: https://tomesphere.com/paper/PMC11847102