Oligonucleotides Hold Promise as a Therapy for Friedreich's Ataxia: Friedreich's ataxia currently is incurable, but synthetic antisense oligonucleotides have demonstrated promising results in increasing frataxin gene expression and restoring it to normal levels

AJMG. Volume176, Issue6 June 2018 Pages 1282-1282 doi:10.1002/ajmg.a.38850

Another important point that was demonstrated in this and previous papers is that oligonucleotides increase the expression of FXN. According to Dr. Corey, “That corrects a fundamental defect in the disease specifically at the gene level, so it is a plausible compound for moving forward.”

However, right now the compounds are in the earliest stages, and more evidence is needed that the treatment is safe and effective. “We need to find animal models to begin testing it, and a company needs to be encouraged to develop it,” Dr. Corey says.

 Oligonucleotides Hold Promise as a Therapy for Friedreich's Ataxia: Friedreich's ataxia currently is incurable, but synthetic antisense oligonucleotides have demonstrated promising results in increasing frataxin gene expression and restoring it to normal levels