Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression

Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression, Negin Mozafari, Salome Isabel Marques Milagres, Cristina Simoes Jesus Rocha, Claudia Marina Vargiu, Fiona Freyberger, Osama Saher, Tea Umek, Pontus Blomberg, Per Trolle Jorgensen, Edvard Carl Igne Smith, Jesper Wengel, Rula L Zain, bioRxiv 2024.01.25.577034; doi:10.1101/2024.01.25.577034 

We examined numerous LNA-DNA AGOs and found that the design, length and their LNA composition have a high impact on the effectiveness of the treatment. Collectively, our results demonstrate the unique capability of specifically designed ONs targeting the GAA:TTC DNA repeats to upregulate FXN gene expression.

Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression