Advancing Treatment for Friedreich’s Ataxia: UMass Lowell’s Study

Muscle Health2

Researchers at UMass Lowell are studying whether a small natural molecule could treat a genetic disorder that causes muscle weakness and progressive nervous system damage. 

Funded by a $313,000 grant from the U.S. Department of Defense, Asst. Prof. Yuho Kim and his team are investigating the potential of a natural molecule, hinokitiol, to alleviate mitochondrial dysfunction associated with the disease.

Friedreich’s ataxia, affecting approximately one in every 50,000 people,

Results from a deficiency of frataxin, a crucial protein for mitochondrial energy production.  Accumulation of iron in the mitochondria leads to oxidative stress and muscle weakness in affected individuals. The team hypothesizes that hinokitiol may restore mitochondrial function by regulating iron levels in skeletal muscle.

The study builds upon previous research by Assoc. Prof. Jonghan Kim, demonstrating hinokitiol’s efficacy in rescuing heart failure and improving skeletal muscle performance.  By targeting mitochondrial dysfunction, the researchers hope to develop a treatment not only for Friedreich’s ataxia but also for other mitochondrial diseases, including cardiac, kidney, diabetes, and cancer.

Through interdisciplinary collaboration, the research team aims to deepen understanding of mitochondrial biology and develop a universal solution for diseases linked to iron overload in the mitochondria.  

Their innovative approach holds promise for transforming treatment strategies and improving outcomes for patients with Friedreich’s ataxia and related disorders.

We would like to thank UMass Lowell for sharing this educational story with us. 

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