Improving Mitochondrial Function To Treat Life-threatening Diseases
Evidence is growing that mitochondrial dysfunction in cells plays a causal or contributory role in many age-related disorders, including Alzheimer’s disease, Parkinson’s disease, diabetes, heart failure and some cancers. In addition, severe mitochondrial dysfunction leads to a variety of deadly childhood diseases (collectively referred to as mitochondrial disease). Recent work in the Kaeberlein lab has provided key insights into therapeutic strategies for treating mitochondrial disease in children.
Funding for next-stage studies would support pre-clinical testing of promising interventions (including small-molecule inhibitors of mTOR signaling) in mammalian models of severe mitochondrial disease. It also would permit Dr. Kaeberlein and his colleagues to develop metabolomic biomarkers that would allow for personalized treatment.
Looking further into the future, the lab will need funding to expand pre-clinical testing to disease models of Parkinson’s disease, Alzheimer’s disease, diabetes and obesity and other conditions associated with mitochondrial dysfunction. Dr. Kaeberlein hopes this work will lead to future clinical trials with children with fatal mitochondrial disease.
Photo by Clare McLean.
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