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The Gene Therapy Center at the 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 R. Jude Samulski, professor of
pharmacology and director of the University's Gene Therapy Center, and 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 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 tissue 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 aim to generate new knowledge about the safety and biological
effectiveness of gene delivery, which will be important in 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 composed of clinical and basic researchers. Along
with Samulski and Boucher, the team includes 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.
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