UT Southwestern Gene Therapy Program
The UT Southwestern Gene Therapy Program seeks to understand the genetic pathology of nervous system disorders and develop treatments for those diseases using gene therapy.
Our researchers focus mainly on diseases with a genetic mutation at the root of disease pathology. A normal copy of a gene or other genetic payload can be packaged into a benign virus called AAV, which acts as a delivery system to transport the gene into the cells of an animal model of the same disease. If the disease improves in the animal model and there are no side effects, this opens the possibility for the experimental treatment to move into a clinical trial after obtaining permission from the FDA.
Program Highlights
- Collaborative research: Our research scientists work in close collaboration with neurologists and other clinical experts at UT Southwestern and Children's Health to understand the underlying pathology of diseases as well as the clinical presentation in patients.
- Translational medicine: Collaboration enables the optimal design of research studies, with the intent to translate what is gained from preclinical (laboratory) studies directly into a clinical trial in humans.
- Design and production: UT Southwestern labs have capabilities for on-site AAV vector production and translational support services, to support preclinical gene therapy studies.
By the Numbers
Faculty
- 14 researchers
Research
- 70+ disease programs
- 5 research labs
About the Program
The UT Southwestern Gene Therapy Program was founded in 2017 under the leadership of Berge Minassian, M.D., and Steven Gray, Ph.D.
Our translational research strives toward a deeper understanding and eventual cure for rare genetic diseases, which carried an economic burden of nearly $1 trillion in 2019 in the U.S. alone, according to a recent EveryLife Foundation for Rare Diseases report.
Much of the current treatment for these neurodegenerative and neurodevelopmental diseases focuses on supportive care and management of symptoms. The UT Southwestern Gene Therapy Program is leading the field with a new wave of transformative treatments targeting the cause of each disease.
Contact Us
Professor
Division Chief, Pediatric Neurology
Jimmy Elizabeth Westcott Distinguished Chair in Pediatric Neurology
