Therapeutic Prospects for Friedreich's Ataxia

Trends Pharmacol Sci. 2019 Apr;40(4):229-233. doi: 10.1016/j.tips.2019.02.001.

Abstract

Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.

Keywords: Friedreich’s ataxia; frataxin; gene therapy; mitochondria; oligonucleotide; oxidative stress.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Disease Progression
  • Friedreich Ataxia / physiopathology
  • Friedreich Ataxia / therapy*
  • Humans
  • Iron-Binding Proteins / metabolism*
  • Mitochondria / pathology*

Substances

  • Iron-Binding Proteins
  • frataxin