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

Saeed Anwar(Shahjalal University of Science and Technology), Toshifumi Yokota(University of Alberta), Umme Sabrina Haque(University of Alberta), Rohini Roy Roshmi(University of Alberta), Anne Bigot(Inserm), Stanley Woo(University of Alberta), William Duddy(University of Ulster), Rika Maruyama(University of Alberta), Joshua J. A. Lee(University of Alberta)
Molecular Therapy — Nucleic Acids
December 21, 2024
Cited by 2


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