Alpha-1 Research Program

Mark Brantly, MD

Mark Brantly, MD, a 1979 graduate of the College of Medicine and director of the college’s Alpha-1 Research Program, has been working for more than a decade to help people suffering from alpha-1 antitrypsin deficiency (AAT), an inherited disorder that can cause lung disease in adults and liver disease in children and adults. Use of the AAV appears to be effective when injected directly into the muscle, and previous studies in animals have been encouraging. Dr. Brantly and his colleagues at the Powell Gene Therapy Center at UF began clinical trials using gene therapy in patients with alpha-1 antitrypsin deficiency in 2004.