An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models. Catherine Gérard, Xiao Xiao, Mohammed Filali, Zoé Coulombe, Marie Arsenault, Jacques Couet, Juan Li, Marie-Claude Drolet, Pierre Chapdelaine, Amina Chikh & Jacques P Tremblay; Molecular Therapy — Methods & Clinical Development 1, Article number: 14044 doi:10.1038/mtm.2014.44
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