For the past 12 years, University of Florida researchers at the Powell Gene Therapy Center have pioneered gene therapies aimed at helping patients with inherited blindness and paralyzing neuromuscular diseases.
With many of these therapies finally about to cross over from the laboratory to mainstream medicine, the Powell Center’s original benefactors, Earl and Christy Powell, of Miami, are making another major push to accelerate help for people with genetic diseases.
Their $5 million gift will establish the Earl and Christy Powell University Chair in Gene Therapy and Genetics Research at the UF Health Science Center and reinforce the couple’s commitment to advancing the field of gene therapy.
The Powells first donated $2 million to UF’s internationally recognized program in gene therapy research in 2000, and the university renamed the center the Powell Gene Therapy Center.
“Earl and Christy Powell’s past generosity has had a significant impact on gene therapy and its translation to patient care,” said UF President Bernie Machen. “Through strong endowments such as those created by the Powells, the university and the Health Science Center are able to recruit and retain the best minds in medicine — scientists and physicians who shape the future of health care.”
The chair in gene therapy and genetics research, to be held by an internationally recognized leader in gene therapy, will focus on the development of methods for safe and effective delivery of therapeutic genes to patients with a variety of diseases.
UF researchers with the Powell Center have been instrumental in the discovery of ways to replace defective genes with healthy ones to treat diseases such as congenital blindness, cystic fibrosis, Pompe disease and alpha-1 antitrypsin deficiency.
Advances include pioneering the use of a harmless virus, called AAV, as a vehicle to deliver replacement genes, the use of gene therapy to improve the ability of patients with Pompe disease to breathe on their own and success in gene therapy clinical trials to reverse a form of blindness known as Leber’s congenital amaurosis.
UF geneticists also are studying the use of gene therapy to deliver cancer-fighting drugs more effectively.
“The discoveries made at UF over the last decade represent concrete examples of the power of collaboration between scientists in the Powell Center with other genetics researchers across campus, including the McKnight Brain Institute, the Emerging Pathogens Institute and many other areas of the university,” said David S. Guzick, M.D., Ph.D., senior vice president for health affairs and president of the UF&Shands Health System. “The Powell chair in gene therapy and genetics research will facilitate and expand these collaborative efforts that have the potential to change the future of health care delivery.”