Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy

Autor: Stephen M. Kaminsky, Laith Alyass, Ronald G. Crystal, Katie M. Stiles, Bishnu P. De, Christiana Salami, Clarisse Jose, Maria J. Chiuchiolo, Dolan Sondhi, Georges-Ibrahim Cisse, Katie Jackson
Rok vydání: 2020
Předmět:
Zdroj: Human Gene Therapy. 31:819-827
ISSN: 1557-7422
1043-0342
Popis: 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
Databáze: OpenAIRE