Article
Partial restoration of mitochondrial dysfunction by AAV-Ant1 protects from dilated cardiomyopathy in Ant1-/- plus mtDNA mutant mice.
Nature communications - 15 Dec 2025
Angelin Alessia, Keller Kierstin, Lu Peiran, Guarnieri Joseph W, Widjaja Gabrielle A, Sule Rasheed, Janssen Kevin, Olali Arnold, Cen Zimu, Carosi Valeria, Beauplan Maina, Murdock Deborah G, Potluri Prasanth, Pei Liming, Wallace Douglas C
Abstract excerpt
Primary mitochondrial disease (PMD) patients manifesting cardiomyopathy are twice as likely to die as other PMD patients. One PMD with cardiomyopathy is caused by null mutations in the heart-muscle isoform of the adenine nucleotide translocator (SLC25A4, ANT1) gene, with the severity of cardiomyopathy mediated by mitochondrial DNA. To optimize strategies for addressing mitochondrial cardiomyopathy, we generated...
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