Article
Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy.
Human gene therapy - 1 Aug 2020
Salami Christiana O, Jackson Katie, Jose Clarisse, Alyass Laith, Cisse Georges-Ibrahim, De Bishnu P, Stiles Katie M, Chiuchiolo Maria J, Sondhi Dolan, Crystal Ronald G, Kaminsky Stephen M
Abstract excerpt
Friedreich's ataxia (FA), an autosomal recessive disorder caused by a deficiency in the expression of frataxin (FXN), is characterized by progressive ataxia and hypertrophic cardiomyopathy. Although cardiac dysfunction is the most common cause of mortality in FA, the cardiac disease remains subclinical for most of the clinical course because the neurologic disease limits muscle oxygen demands. Previous FXN...
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