Muscular dystrophy (MD) is a group of diseases that weakens and degenerates skeletal muscles. Currently, there is no specific treatment to stop or reverse any of the diseases in MD. Dr. Hua Zhu, PhD, hopes to find a novel method that reskills muscle by improving muscle function.
Dr. Zhu was funded $1,644,080 for his study, "Regulation of Mitsugumin 29 expression in muscle physiology and diseases."
The R01 grant will extend over five years and is allocated to various approaches that examine mitsugumin29 (MG29). The first one-to-two years of the study will focus on targeting the biological role of MG29 in development of muscular dystrophy. From the third-to-fifth year, Dr. Zhu and his team will utilize an animal model before techniques are applied to human patient samples. The animal model consists of transgenic and knockout mice to secure preliminary data for human study application.
From his past experience, Dr. Zhu is very confident that he will find a mechanism to explain why MG29 is reduced in skeletal muscle derived from muscular dystrophy animals and patients. His goal is to find an effective way to regulate MG29 expression, which will essentially improve muscle function. Importantly, MG29 has been linked to muscle function. Within the muscle lacking MG29, there is reduced calcium signaling and increased tiredness during exercise. In the mouse models, mice demonstrated phenotypic symptoms of premature aging due to reduced muscle function. This line of research demonstrates how muscle function improvement equates a better full-body performance.
Most importantly, Dr. Zhu’s project will improve the way healthcare providers treat irreversible diseases disciplines such as MD.
“We will be able to increase people’s daily activity. At the end of [MD], people have to be put in a wheelchair. If we can improve muscle function, people affected by the disease will be able to lead more of a normal life,” says Dr. Zhu.
Dr. Zhu’s interest in the subject was discovered during his post-doctoral training. During the training process, Dr. Zhu was experimenting with metabolic disease, which is also involved in compromised muscle function. In a study, he used muscle samples derived from different animal models and found that the muscular dystrophic muscle samples contained a reduced level of MG29. From that point forward, Dr. Zhu commenced his program and examined the molecular mechanisms underlying reduced MG29 in MD muscles.