Powering Up Mitochondrial Functions to Treat Mitochondrial Disease
Technical Report,30 Sep 2016,29 Sep 2017
Childrens Hospital of Philadelphia Philadelphia United States
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We proposed that induction of the ERRalphagamma signaling pathway can enhance mitochondrial function in both cell and animal models of mitochondrial disease. Our major findings include1 We recently compared in detail the different mitochondrial disease animal models under review in Cell Metabolism. We found that the compound Ant1--ND6 mutant mouse model exhibited the earliest and strongest mitochondrial cardiomyopathy phenotype and therefore provided the best therapeutic window for our proposed intervention research strategy.2 We discovered that GDF15 is a heart-derived hormone whose serum level correlates positively with the severity of mitochondrial cardiomyopathy recently published with DOD grant support acknowledged, and it can be used as a biomarker in our studies.