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Future Prospects of Gene Therapy for Friedreich's Ataxia

A plausible therapeutic approach for Friedreich's ataxia is gene therapy. Indeed, Friedreich's ataxia mouse models have been treated with viral vectors encoding 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. This paper reviews the state of the art of gene therapy in Friedreich's ataxia, addressing the main challenges and the most feasible solutions for them.

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