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

Saeed Anwar, Rohini Roy Roshmi, Stanley Woo, Umme Sabrina Haque, Joshua James Arthur Lee, William John Duddy, Anne Bigot, Rika Maruyama, Toshifumi Yokota

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology