Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts

Khonsari H , Schneider M , Al-Mahdawi S , Chianea YG , Themis M , Parris C , Pook MA , Themis M; Gene Therapy accepted article preview 12 August 2016; doi: 10.1038/gt.2016.61

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 Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts