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- Title
Future Prospects of Gene Therapy for Friedreich's Ataxia.
- Authors
Ocana-Santero, Gabriel; Díaz-Nido, Javier; Herranz-Martín, Saúl; Köster, Reinhard
- Abstract
Friedreich's ataxia is an autosomal recessive neurogenetic disease that is mainly associated with atrophy of the spinal cord and progressive neurodegeneration in the cerebellum. The disease is caused by a GAA-expansion in the first intron of the frataxin gene leading to a decreased level of frataxin protein, which results in mitochondrial dysfunction. Currently, there is no effective treatment to delay neurodegeneration in Friedreich's ataxia. A plausible therapeutic approach is gene therapy. Indeed, Friedreich's ataxia mouse models have been treated with viral vectors en-coding for either FXN or neurotrophins, such as brain-derived neurotrophic factor showing promising results. Thus, gene therapy is increasingly consolidating as one of the most promising therapies. However, several hurdles have to be overcome, including immunotoxicity and pheno-toxicity. We review the state of the art of gene therapy in Friedreich's ataxia, addressing the main challenges and the most feasible solutions for them.
- Subjects
FRIEDREICH'S ataxia; GENE therapy; BRAIN-derived neurotrophic factor; EXPRESSIVE arts therapy; FRATAXIN; NEUROTROPHIN receptors
- Publication
International Journal of Molecular Sciences, 2021, Vol 22, Issue 4, p1815
- ISSN
1661-6596
- Publication type
Article
- DOI
10.3390/ijms22041815