Translational Research Recognized

MD-PhD student hopes gene therapy discoveries will help children with Duchenne muscular dystrophy

For those who suffer from debilitating genetic disorders, the rapidly developing world of gene therapy research offers new hope for a cure. Three years ago, the seemingly limitless potential of this research led medical student Brian Bostick to study how to deliver missing genes to patients.

"Gene therapy is a technology that opens the door for treatment of a variety of diseases," Bostick said. "It could allow us to target a disease at the level where the defect is located, which is very exciting."

Brian Bostick

Bostick, an MD-PhD graduate student at the University of Missouri, was selected to present his research at the annual meeting of the American College of Physicians (ACP) in April 2010 in Toronto. Bostick's research focuses on finding treatment for a disease that has no known cure — Duchenne muscular dystrophy (DMD). His entry was one of two chosen for oral presentation among 32 entries in the basic science category of the ACP National Medical Students Abstract Competition.

Due to progressive muscle deterioration, children with muscular dystrophy become paralyzed and usually die of respiratory or cardiac failure before their 30th birthday. Approximately 250,000 people in the United States have some form of muscular dystrophy, and DMD is the most common type of the disease, predominantly affecting males.

Bostick has been studying under the guidance of Dongsheng Duan, PhD, Margaret Proctor Mulligan Distinguished Professor in Medical Research in the Department of Molecular Microbiology and Immunology and recipient of the 2009 Chancellor's Award for Outstanding Research and Creative Activity.

"Brian's muscular dystrophy work serves as an outstanding example of the kind of translational research training we are giving our MD-PhD students," Duan said. "We are really working to translate basic science research into the clinical practice, and I believe that is going to change the thinking on how many diseases are treated."

Duan is leading research to develop a new therapy delivery method (viral gene therapy) for DMD patients, which he has tested in mice and now dogs. Unlike other methods, Duan's is effective for both skeletal muscles, such as those in the arms and legs, and cardiac muscles. It has been shown to reach every muscle in large animals.

Bostick is a student in the MU Molecular Microbiology & Immunology – Veterinary Pathobiology Graduate Program and a student in the Tom and Anne Smith MD-PhD Program. He plans to complete a residency in internal medicine at the MU School of Medicine and a postdoctoral research fellowship.