Defining minimal expression levels for normal cell function in different tissues, including brain, will be needed to develop effective therapies. These experiments are an important contribution to the field of FA, because they define toxicity of FXN overexpression and, more importantly, identify mechanisms underlying this toxicity.
Monday, March 7, 2022
Gene therapy for Friedreich ataxia: Too much, too little, or just right?
R. Mark Payne, Molecular Therapy - Methods & Clinical Development, Volume 25, 2022, Pages 1-2, doi:10.1016/j.omtm.2022.02.008
