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

Brahim Belbellaa, Laurence Reutenauer, Nadia Messaddeq, Laurent Monassier, Hélène Puccio; Molecular Therapy - Methods & Clinical Development, 2020, doi:10.1016/j.omtm.2020.08.018.

this study underlines the need, during the development of gene therapy approaches, to consider appropriately vector expression level, long term safety and biomarkers to monitor such events.

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