UNC-Chapel Hill gene therapy center awarded $9.2 million from NIH

December 06, 2001

CHAPEL HILL - The Gene Therapy Center at the University of North Carolina at Chapel Hill School of Medicine will receive $9.2 million over five years from the National Heart, Lung and Blood Institute of the National Institutes of Health.

The center will use the funds to help translate basic research knowledge of gene delivery into safe and rigorous human clinical trials in lung and blood disorders. The ultimate goal is to provide new treatments for diseases caused by single-gene defects such as cystic fibrosis and hemophilia.

Co-principal investigators for the program are Dr. R. Jude Samulski, professor of pharmacology and director of the university's Gene Therapy Center, and Dr. Richard Boucher, director of the Cystic Fibrosis Center and the department of medicine's pulmonary division.

Samulski helped pioneer the use of genetically altered adeno-associated viruses to deliver genes into the body. Molecular studies in his UNC laboratory have uncovered important factors that could contribute to the success or failure of human gene therapy using altered viruses.

Boucher has published more than 300 articles on cystic fibrosis and gene therapy. In 1992, he helped develop a gene "knock-out" mouse model for cystic fibrosis. His laboratory continues its major interest in the functions of airway epithelia in health and disease.

"This award speaks to our basic research capability and our unique strength in animal models. It also acknowledges the importance of understanding the basic science first before moving into the clinical setting," Samulski said. Two program projects will be aimed at understanding and overcoming inefficient gene delivery related to virus entry into cells and the persistence of expression of the transferred gene. These studies are aimed at generating new knowledge about the safety and biological efficacy of gene delivery, which will be of importance to the design of future clinical trials.

Two additional projects will investigate the cell biology of target tissue and will study animal models for airway and hemophilia disorders. These will be aimed at increasing the access of transferred genes to airway tissue and developing novel models, including a 'humanized' hemophilia mouse.

The investigative team is comprised of clinical and basic researchers. Along with Samulski and Boucher, the team includes Drs. Darrel Stafford, Tal Kafri, John Olsen, Paul Monahan, Ray Pickles, Johnny Carson, and Barbara Grubb. "We have a unique interactive team. We have the people who can provide different gene delivery systems and develop novel animal models," Samulski said.
Media note: Contact Dr. Samulski at 919-962-3285, rjs@med.unc.edu. School of Medicine contact, Les Lang, 919-843-9687, llang@med.unc.edu

University of North Carolina Health Care

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