Careers  |  Sign In  |  Register  |   Twitter

Gene Therapy Trials for Rare Diseases on a Small Scale at UT Southwestern

At UT Southwestern Medical Center, Steven Gray, PhD, develops gene therapies for ultra-rare diseases, such as Leigh syndrome. Dr. Gray mentions that because gene therapy is a disruptive technology — a one-time treatment with a potential life-long benefit — it doesn’t fit the traditional pharmaceutical business model which makes it difficult to attract the pharmaceutical industry’s support. Conducting small clinical trials for these treatments at an academic medical center allows researchers to move their experimental therapies forward in the development process. It also gives hope to those living with a very rare, and currently incurable, disease.

Click here to learn more about this development at UT Southwestern Medical Center.