Dawn Cornelison is on a mission to counteract the effects of aging, the effects of muscular dystrophy, and other neuromuscular diseases. The assistant professor of Biological Sciences must first find answers to the crucial questions regarding the robust nature of muscle regeneration.
When doing research, Cornelison says, “you have to have a pretty high tolerance for failure bordering on extreme stubbornness… You’ve got to be able to live with not getting things to work all the time.” All of her research is funded by external grants, which means she has to secure external funding in order to pay her fellow researchers, house the lab’s mice, or buy materials. Currently, Cornelison is receiving funding from the National Institutes of Health and the Muscular Dystrophy Association.
As a graduate student in the Department of Molecular Microbiology and Immunology in MU’s School of Medicine, Brian Bostick works with professor Dongsheng Duan in the area of gene therapy. Bostick’s research seeks to develop a treatment for the most common form of muscular dystrophy, Duchenne muscular dystrophy, in which patients are missing a gene called dystrophin. Gene therapy involves the replacement or addition of a missing gene. Bostick’s research involves inserting this gene into a virus and then injecting it into an animal body. “Just by using the normal properties of how a virus works,” Bostick explains, “we can actually replace genes that are missing.”
Bostick’s research focuses specifically on the heart disease associated with Duchenne muscular dystrophy, where a gradual weakening of the muscles occurs—starting with the larger muscles—so that patients have trouble breathing by the time they are teenagers. For a long time, such respiratory problems had been the major cause of death among DMD patients, but doctors are now better able to treat the respiratory disease. Because the heart muscle also needs dystrophin to function properly, heart disease worsens as these patients live longer. Heart disease, in fact, is now a major cause of death among DMD patients, a problem that Bostick and his mentor Duan seek to address by developing a heart disease model in mice.
Bostick offers a quick tour of Duan’s laboratory, illustrating the processes involved in several research projects—from the mouse treadmill to the surgical area and where the mice are kept under observation. Delicately selecting several mice, Bostick shows examples of a normal mouse, one with MD, and another with MD undergoing gene replacement therapy. The difference, in both size and activity, between the untreated mouse and the one given gene therapy is remarkable and promising for future applications of this research.