Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation. Yameogo, Pouiré et al. Molecular Therapy Nucleic Acids, Volume 0, Issue 0, 102617. DOI:10.1016/j.omtn.2025.102617
This ASO strategy may be therapeutically feasible for FRDA patients with other point mutations that cause splicing defects. Success in developing treatments for disorders with only a few known cases will give hope to FRDA patients carrying these rare point mutations.
Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation
