51视频

12/20/2023
By Karen Angelo

Researchers at 51视频 are studying whether a small natural molecule could treat a genetic disorder that causes muscle weakness and progressive nervous system damage.听

Friedreich鈥檚 ataxia affects about one in every 50,000 people. This disease leads to a loss of coordination, difficulty walking and poor balance. It鈥檚 caused by a deficiency of frataxin, a protein that is crucial for generating energy in the mitochondria, the powerhouses of cells.听

Asst. Prof. Yuho Kim of the Department of Physical Therapy and Kinesiology was awarded a $313,000 grant from the U.S. Department of Defense to study the key mechanism and treatment in the pathological development of Friedreich鈥檚 ataxia in muscles.听

鈥淭his grant helps extend my ongoing research into studying how the mitochondria change molecular signaling, function and structure in cardiac and skeletal muscle disease conditions,鈥� says Kim, who is collaborating with Assoc. Prof. Jonghan Kim of the Department of Biomedical and Nutritional Sciences and Prof. and Assoc. Dean for Research and Graduate Studies Dhimiter Bello of the Zuckerberg College of Health Sciences.听

鈥淣ow, we will further understand the roles of mitochondria in muscle health and diseases,鈥� he adds.听

The research team will study the potential ability of a small natural molecule called hinokitiol to correct mitochondrial dysfunction in the skeletal muscle of Friedreich鈥檚 ataxia. For the mitochondria to work effectively, the body needs the right balance of iron. In people with this ataxia, however, iron accumulates, which causes mitochondrial dysfunction by oxidative stress.听

The researchers will test how well hinokitiol regulates the level of iron in the mitochondria and improves muscle weakness in Friedreich鈥檚 ataxia.听

鈥淚f successful, our proposed treatment could be effective in other mitochondrial diseases, such as cardiac, kidney, diabetes and cancer,鈥� says Kim. 鈥淥ur findings will help deepen our understanding of mitochondrial biology in muscle health and disease conditions where mitochondria play a major role.鈥澛�

Jonghan Kim, who is an expert on iron disorders, demonstrated in earlier research that the molecule hinokitiol can rescue heart failure caused by doxorubicin, a widely used cancer drug. The results also suggested that the same molecule can be used to improve the performance of skeletal muscle.听

鈥淥ur research team at 51视频, with complementary expertise and talents, can provide a universal solution for diseases caused by or worsened by iron overload in the mitochondria,鈥� he says. 鈥淎lthough two different health problems occur in different organs and by different mechanisms, the dysfunction is from one common physiological defect 鈥� impaired mitochondrial function that is controlled by iron.鈥�