What is Gene Therapy?
The goal of gene therapy is to insert genetic material into cells in the body, usually to correct a gene that is missing or faulty. Gene therapy can also change the degree to which a gene is expressed, or potentially “silence” the expression of a disease-causing gene. Gene therapy opens up enormous possibility to transformatively treat diseases which are caused by a known genetic mutation. "What is Gene Therapy?" from U.S. Food & Drug Administration
What makes UT Southwestern Gene Therapy Program Unique?
The Gene Therapy Program at UT Southwestern connects researchers across several academic departments with clinicians to understand the underlying pathology of disease as well as the clinical presentation in patients. This 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. UT Southwestern has built an on-site vector core facility, which is fully capable of manufacturing the viral delivery systems used in both research studies and clinical trials.
The UT Southwestern Gene Therapy program aims to understand the molecular and cellular mechanisms of genetic disorders, and leverage this knowledge into preclinical animal models with the ultimate goal of clinical trials leading to approved treatments in humans. This translation 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 are supportive care and management of symptoms. The UT Southwestern Gene Therapy Program envisions a new wave of transformative treatments targeting the cause of each disease.