UT Southwestern establishing Wellstone Muscular Dystrophy Center to further research for new treatments using gene-editing technique
Dr. Eric Olson, Chairman of Molecular Biology and Director of the Hamon Center for Regenerative Science and Medicine, will co-direct the Wellstone Muscular Dystrophy Center with Dr. Pradeep Mammen, Associate Professor of Internal Medicine and Medical Director of UT Southwestern’s Neuromuscular Cardiomyopathy Clinic.
“UT Southwestern is the perfect environment for the Wellstone Center. We can merge cutting-edge science with clinical application,” said Dr. Olson, who holds the Robert A. Welch Distinguished Chair in Science, the Pogue Distinguished Chair in Research on Cardiac Birth Defects, and the Annie and Willie Nelson Professorship in Stem Cell Research.
Wellstone Centers of Excellence work to translate scientific findings and technological developments into novel treatments for muscular dystrophy, and to promote basic, translational, and clinical research.
“The NIH’s investment in Dr. Olson’s novel approach to gene editing significantly enhances the potential to have a profound impact on the course of this devastating disease,” said Dr. Daniel K. Podolsky, President of UT Southwestern, who holds the Philip O’Bryan Montgomery, Jr., M.D. Distinguished Presidential Chair in Academic Administration, and the Doris and Bryan Wildenthal Distinguished Chair in Medical Science. “In developing these innovative gene-editing techniques, Dr. Olson and his colleagues reflect the bright promise of the Hamon Center for Regenerative Science and Medicine.”
UT Southwestern’s Wellstone Center will focus on Duchenne muscular dystrophy, an inherited form of muscular dystrophy that is characterized by progressive muscle degeneration and weakness. It usually presents itself in boys age 3 to 5 and often leads to death by the early 30s. The disorder is caused by a mutation in the gene dystrophin, or DMD, which creates the protein dystrophin that helps keep muscle cells intact.
The UTSW Wellstone Center will tackle two main scientific projects, along with patient education and student training. One project will seek to correct muscular dystrophy in a mouse model of Duchenne muscular dystrophy using a gene-editing technique developed at UT Southwestern called Myoediting. The other project will use blood cells from Duchenne muscular dystrophy patients to create stem cells, called induced pluripotent stem cells (iPSCs), which can be used for Myoediting to delete the DMD mutation in the patient’s cells. The goal of the UTSW Wellstone Center is to eventually take potential therapies developed from this gene-editing technique to human clinical trials.
“Our challenge is to translate this basic science discovery into an effective clinical therapy to improve the lives of patients with Duchenne muscular dystrophy,” said Dr. Mammen.
Leadership of the UTSW Wellstone Center is comprised of clinicians and scientists from a variety of disciplines:
- Dr. Susan Iannaccone, Professor and Chief of the Division of Child Neurology in the Department of Pediatrics, and of Neurology and Neurotherapeutics, who holds the Jimmy Elizabeth Westcott Distinguished Chair in Pediatric Neurology, will serve as Associate Director of the UTSW Wellstone Center.
- Dr. Rhonda Bassel-Duby, Associate Director of the Hamon Center for Regenerative Science and Medicine, Professor of Molecular Biology, will serve as the Co-Principal Investigator of the Myoediting project with Dr. Olson.
- Dr. Jay Schneider, Associate Director of the Hamon Center for Regenerative Science and Medicine, Associate Professor of Internal Medicine, and holder of the Dallas Heart Ball Chair in Cardiac Research, will serve as Co-Principal Investigator for the pluripotent stem cells project, along with Dr. Mammen.
- Dr. Jaya Trivedi, Professor of Neurology and Neurotherapeutics, and Dr. Beverly Rothermel, Associate Professor of Internal Medicine and Molecular Biology, will lead the patient education and student training.
Approximately 20 clinicians and scientists from multiple disciplines at UT Southwestern will be involved in the UTSW Wellstone Center initially, with plans to expand faculty participation.
Funding for the Wellstone Center totals $7.8 million over five years from the National Institute of Child Health and Human Development, and the National Heart, Lung, and Blood Institute, both institutions with the National Institutes of Health. UT Southwestern joins five other Wellstone Centers, including the University of Florida, University of Iowa, University of Massachusetts Medical Center, University of Rochester, and the University of Washington.
UT Southwestern’s Hamon Center for Regenerative Science and Medicine was made possible by a $10 million endowment gift from the Hamon Charitable Foundation. The Center’s goal is to understand the basic mechanisms for tissue and organ formation, and then to use that knowledge to regenerate, repair, and replace tissues damaged by aging and injury.
About UT Southwestern Medical Center
UT Southwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institution’s faculty includes many distinguished members, including six who have been awarded Nobel Prizes since 1985. The faculty of more than 2,700 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide medical care in 40 specialties to about 92,000 hospitalized patients and oversee approximately 2.1 million outpatient visits a year.