Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells
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Authors
- Naoki Suzuki (ORCID: https://orcid.org/0000-0001-8880-8554)
Institutions
- Tohoku University (JP)
- KKR Tohoku Kosai Hospital (JP)
Publication Details
- Journal
- Molecular Therapy — Nucleic Acids
- Published
- 2025-02-04
- DOI
- https://doi.org/10.1016/j.omtn.2025.102462
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00