Associated Papers
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells.
Anwar S et al.
Molecular Therapy. Nucleic Acids, 2025
Dysferlin-deficient immortalized human myoblasts and myotubes as a useful tool to study dysferlinopathy.
Philippi S et al.
Plos Currents, 2012
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