Indiana University To Host National Biorepository For Gene Therapy

Main Category: Genetics
Also Included In: Biology / Biochemistry
Article Date: 20 Jun 2008 - 3:00 PDT

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Indiana University School of Medicine will be home to the nation's sole National Gene Vector Biorepository and Coordinating Center (NGVB) for gene therapy research with a three-year, $3 million grant from the National Institutes of Health.

The program is directed by Kenneth Cornetta, M.D, Joe C. Christian Professor and chair of Medical and Molecular Genetics and professor of medicine and of microbiology and immunology. The three year grant to the School of Medicine was effective June 15.

"This center will continue the IU School of Medicine's history of providing critical resources to gene therapy researchers, who are working to find treatments for some of our most difficult diseases," said Dr. Cornetta.

Researchers use gene vectors, such as disabled viruses, to carry genetic materials in the body in hopes of treating or preventing disease. For example, genes important for fighting infections may be inserted into immune system cells that have been rendered inactive by genetic mutations.

The NGVB at Indiana University will help university scientists share research information and substances to promote discoveries, patient safety and compliance with regulations of the Food and Drug Administration. The NGVB's services will include maintaining a warehouse of important reagents, disseminating the results of safety studies, and the storage of biological materials and patient specimens in accordance with FDA regulations.

The IU center will also conduct studies of the safety of various gene therapy techniques, said Dr. Cornetta.

"The new biorepository and center will help leverage information-sharing and expertise across the gene therapy research community," said Barbara M. Alving, M.D., director of the National Center for Research Resources, the NIH center that is funding the grant. "By coordinating preclinical studies, this resource will maximize NIH's investment in gene vector research and help translate new knowledge into tangible benefits for patients."

Scientists are using gene transfer techniques in studies involving a broad range of diseases including cancer, heart disease, cystic fibrosis, immune disorders including arthritis, and infectious diseases. An NIH database lists more than 500 such trials currently under way.

The NGVB replaces the National Gene Vector Laboratory and Coordinating Center at the IU School of Medicine, which provided gene therapy vectors for use in clinical trials. Indiana University continues to provide gene therapy vectors under contracts with the National Cancer Institute and the National Heart Lung and Blood Institute.

Indiana University School of Medicine