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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, Willium John Duddy,Vincent Mouly,Rika Maruyama,Toshifumi Yokota

Molecular Therapy - Nucleic Acids(2024)

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