UT Southwestern has been awarded a $7.8 million, five-year grant to establish the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, with a special focus on inherited Duchenne muscular dystrophy (DMD).
“UT Southwestern is the perfect environment for the Wellstone Center. We can merge cutting-edge science with clinical application,” said Dr. Eric Olson, who will co-direct the new center with Pradeep Mammen, in a recent press release.
The Wellstone Center will start with two scientific projects while also investing in student training and patient education. One project will try to correct muscular dystrophy in a DMD mouse model using a gene-editing technique developed at UTSW and known as myoediting. The other will create stem cells from the blood cells of DMD patients in a process called induced pluripotent stem cells (iPSCs) that can be used for myoediting to delete the DMD mutation in the patients’ cells. UTSW Wellstone Center ultimately wants to advance the potential therapies developed from this gene-editing technique into clinical trials.
“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 Daniel K. Podolsky, MD and president of UT Southwestern. “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.”
Other Wellstone Center members are Dr. Rhonda Bassel-Duby, co-principal investigator of the myoediting project with Dr. Olson; Dr. Mammen, who also serves as co-principal investigator for the pluripotent stem cells project alongside Jay Schneider; and Susan Iannaccone, associate director of the UTSW Wellstone Cente.
“Our challenge is to translate this basic science discovery into an effective clinical therapy to improve the lives of patients with Duchenne muscular dystrophy,” concluded Dr. Mammen.
UTSW Wellstone Center funding comes from the National Institute of Child Health and Human Development, and the National Heart, Lung, and Blood Institute, both part of the National Institutes of Health. UT Southwestern is one of six academic institutions with Wellstone Centers, with others at the University of Florida, University of Iowa, University of Massachusetts Medical Center, University of Rochester, and the University of Washington.
Paul Wellstone was a respected U.S. senator from Minnesota, who died in a plane crash in October 2002.