Mol Ther Methods Clin Dev. 2022 Mar 4;25:1-2. doi: 10.1016/j.omtm.2022.02.008. eCollection 2022 Jun 9.
This is an editorial commenting on the article by Huichalaf et al. “In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency.” published in Molecular Therapy: Methods & Clinical Development.
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Gene therapy for Friedreich ataxia: Too much, too little, or just right?
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