BETHESDA, MD, June 13, 2003 Researchers at the National Human Genome Research Institute (NHGRI) may have taken a major step towards safer gene therapy for patients. In a report in this week's issue of Science, a research team from NHGRI's Division of Intramural Research demonstrates for the first time that the genetically engineered mouse virus used in gene therapy trials tends to insert itself at the beginning of genes in the target cell, potentially disrupting the genes normal function.

"Gene therapy is a promising field that offers fundamentally new ways of curing human illness," said NHGRI Director Francis S. Collins, M.D., Ph.D. "A decade of clinical studies has demonstrated the complexity of the biology behind gene therapy and the technical problems researchers have experienced. This study provides insight into one of the most serious, current technical hurdles."

The discovery may lead to safer gene therapy techniques.

"Provided this new insight, researchers can now aim to improve the design of gene therapy techniques that can insert genes in less risky areas of the genome," said NHGRI Scientific Director Eric D. Green, M.D., Ph.D., who is director of the Division of Intramural Research and chief of the Genome Technology Branch where the work was performed. "The viruses used therapeutically can be altered in various ways and now tested in the lab to see if they insert curative genes more safely."

In January 2003, the U.S. Food and Drug Administration (FDA) placed a "clinical hold" on 27 gene therapy studies after two children being treated by French researchers for a form of severe combined immunodeficiency disease developed a leukemia-like condition.

Most current gene therapy experiments attempt to cure genetic illnesses by inserting normal, functional copies of a gene into target cells of the body. Researchers have learned to genetically engineer different types of viruses so they can infect target cells
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Contact: Geoff Spencer
spencerg@mail.nih.gov
301-402-0911
NIH/National Human Genome Research Institute
12-Jun-2003

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