A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins

Por Chapdelaine, Pierre, Coulombe, Zoé, Chikh, Amina, Gérard, Catherine, Tremblay, Jacques P
Publicado em 2013

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

Por Gérard, Catherine, Xiao, Xiao, Filali, Mohammed, Coulombe, Zoé, Arsenault, Marie, Couet, Jacques, Li, Juan, Drolet, Marie-Claude, Chapdelaine, Pierre, Chikh, Amina, Tremblay, Jacques P
Publicado no Mol Ther Methods Clin Dev (2014)