Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression
Negin Mozafari, Salomé Milagres, Tea Umek, Cristina S. J. Rocha, Claudia Marina Vargiu, Fiona Freyberger, Osama Saher, Марек Напиерала, Jill S. Napierala, Pontus Blomberg, Per T. Jørgensen, Tanel Punga, C. I. Edvard Smith, Jesper Wengel, Rula Zain
This study shows that anti-gene oligonucleotides targeting GAA*TTC repeats in Friedreichs ataxia increase FXN expression in patient cells.
Molecular Therapy — Nucleic Acids · Apr 17, 2025Peer-reviewedMolecular Biology