May Revolutionize Therapy in Muscular Dystrophy and
Other Neurodegenerative Disorders
Newswise — New Brunswick, NJ – Scientists at UMDNJ-Robert Wood Johnson Medical School are a step closer to treating, and perhaps preventing, muscle damage caused by neurodegenerative disorders and other forms of disease. In a newly published study, released today and cited as a Paper of the Week by the Journal of Biological Chemistry, the team has discovered that the gene polymerase I and transcript release factor, or PTRF, is an essential component of the cell process that repairs damaged muscle tissue. This discovery has the potential to lead to development of therapeutic treatment for patients who suffer from severe complications of diseases such as muscular dystrophy, cardiovascular disorders and other degenerative conditions.
The research was led by Jianjie Ma, PhD, professor and acting chair of physiology and biophysics at UMDNJ-Robert Wood Johnson Medical School. Hua Zhu, PhD, an instructor in Dr. Ma’s laboratory, is the first author on the manuscript describing this discovery.
According to Dr. Ma, human cells are continuously injured and naturally repaired throughout their life span. For instance, micro tears can occur as muscles contract within the body during normal everyday activities. However, diseases such as diabetes, cardiovascular disorders and muscular dystrophy, and even aging, compromise the method through which the body repairs its own tissues, resulting in severe damage. His research team announced in December 2008 that it had discovered MG53 as a key initiator of membrane repair in damaged tissue, making it the first group to specifically pinpoint a protein responsible for promoting cell repair.
In this new study, the team’s research has revealed that in order for MG53 to successfully repair damaged tissue, it must work cooperatively with PTRF. During the muscle repair process PTRF acts as a docking protein, potentially binding MG53 with exposed membrane cholesterol at the injury site. When PTRF is absent in cells, the binding process is interrupted and MG53 can not successfully repair damaged tissues.
“The identification of PTRF as a molecule that anchors MG53 to injured tissue will help us to better understand how a cell membrane can repair itself,” said Dr. Ma. “The discovery of PTRF as a necessary component in the initiation of muscle repair is another hopeful step toward the development of therapeutic treatments for patients with muscle and cardiovascular disease.”
The research was supported by grants from the National Institutes of Health. The paper may be found online at: http://www.jbc.org/content/286/15/e99924.full. A subscription may be required for access.
About UMDNJ-Robert Wood Johnson Medical School
As one of the nation’s leading comprehensive medical schools, UMDNJ-Robert Wood Johnson Medical School is dedicated to the pursuit of excellence in education, research, health care delivery, and the promotion of community health. In cooperation with Robert Wood Johnson University Hospital, the medical school’s principal affiliate, they comprise New Jersey’s premier academic medical center. In addition, Robert Wood Johnson Medical School has 34 other hospital affiliates and ambulatory care sites throughout the region.
As one of the eight schools of the University of Medicine and Dentistry of New Jersey with 2,800 full-time and volunteer faculty, Robert Wood Johnson Medical School encompasses 22 basic science and clinical departments, hosts centers and institutes including The Cancer Institute of New Jersey, the Child Health Institute of New Jersey, the Center for Advanced Biotechnology and Medicine, the Environmental and Occupational Health Sciences Institute, and the Stem Cell Institute of New Jersey. The medical school maintains educational programs at the undergraduate, graduate and postgraduate levels for more than 1,500 students on its campuses in New Brunswick, Piscataway, and Camden, and provides continuing education courses for health care professionals and community education programs. To learn more about UMDNJ-Robert Wood Johnson Medical School, log on to rwjms.umdnj.edu. Find us online at www.Facebook.com/RWJMS and www.twitter.com/UMDNJ_RWJMS.