In Vivo Overexpression of Frataxin Causes Toxicity Mediated by Iron-Sulfur Cluster Deficiency.
Molecular therapy Methods & clinical development（2022）
关键词
Friedreich's ataxia,gene therapy,adeno-associated virus,frataxin,iron-sulfur cluster,mitochondria,toxicity,liver regeneration,transgene overexpression,loss of transgene expression
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