Article

High levels of frataxin overexpression leads to mitochondrial and cardiac toxicity in mouse models

2020-04-01

Abstract excerpt

<h4>ABSTRACT</h4> Friedreich ataxia (FA) is currently an incurable inherited mitochondrial disease caused by reduced levels of frataxin (FXN). Cardiac dysfunction is the main cause of premature death in FA. AAV-mediated gene therapy constitutes a promising approach for FA, as demonstrated in cardiac and neurological mouse models. While the minimal therapeutic level of FXN protein to be restored and biodistributio...

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Literature Corpus work: 6695fbcc-3fec-5d1e-a7be-9e33bffb54f7
DOI: 10.1101/2020.03.31.015255

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