Associated Papers
Molecular Mechanisms and Therapeutics for the GAA·TTC Expansion Disease Friedreich Ataxia.
Gottesfeld JM
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics, 2019
Highly specific ubiquitin-competing molecules effectively promote frataxin accumulation and partially rescue the aconitase defect in Friedreich ataxia cells.
Rufini A et al.
Neurobiology of Disease, 2015
Discovery of a novel small molecule inhibitor targeting the frataxin/ubiquitin interaction via structure-based virtual screening and bioassays.
Lavecchia A et al.
Journal of Medicinal Chemistry, 2013
Preventing the ubiquitin-proteasome-dependent degradation of frataxin, the protein defective in Friedreich's ataxia.
Rufini A et al.
Human Molecular Genetics, 2011
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