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Restoration of Dysferlin After Exon 32 Skipping in Patient Cells

Florian Barthélémy1,2, Sébastien Courrier3, Marc Bartoli4

  • 1Microbiology Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, CA, USA.

Methods in Molecular Biology (Clifton, N.J.)
|July 28, 2025
PubMed

Keywords:
DysferlinExon skippingMuscular dystrophiesRNA editingTherapies

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