Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression
Negin Mozafari, Salomé Milagres, Tea Umek…·Rula Zain
This study shows that anti-gene oligonucleotides targeting GAA*TTC repeats in Friedreichs ataxia increase FXN expression in patient cells.
6 citations