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

Therapeutic Prospects for Friedreich's Ataxia.

Author information

1
Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham (UAB), 1825 University Boulevard, Birmingham, AL 35294, USA.
2
Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham (UAB), 1825 University Boulevard, Birmingham, AL 35294, USA. Electronic address: jsbutler@uab.edu.

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

PMID:
30905359
DOI:
10.1016/j.tips.2019.02.001
[Indexed for MEDLINE]

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