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Researchers explore gene treatment to obliterate HIV before it does damage

STANFORD, Calif. - Doctors may someday have a new way to combat AIDS by going straight to the source: destroying the virus before it has a chance to wreak havoc on a patient's immune system.

Thomas Merigan, MD, the George and Lucy Becker Professor of Medicine in infectious diseases at Stanford University School of Medicine, is seeking volunteers for a study to test a possible method of empowering an infected person's own cells to destroy HIV as it enters the cell. The process involves removing the patient's stem cells - the ones in the bloodstream that form the different immune system cell types that HIV infects, such as T cells and macrophages - and inserting a gene that produces an HIV-obliterating enzyme.

"This is a broad-spectrum treatment that could integrate well with other therapies as the disease progresses, as it will in all patients eventually," said Merigan, who is collaborating with other researchers at UCLA and St. Vincent's Hospital in Sydney, Australia, under the sponsorship of Johnson & Johnson Research. "The goal is to genetically engineer the cells and make them resistant to infection."

The strategy developed by Merigan and his colleagues relies on the fact that the genetic information of HIV is encoded in RNA rather than DNA. Enzymes called ribozymes can chew up RNA at very specific sites, rendering it inactive. If a ribozyme specific to HIV RNA were present inside the cells that the virus infects, then it could constitute a first-line defense against the invasion. Even if HIV did make it into the cell and replicated, the ribozyme could potentially cleave the HIV RNA at various steps during the virus' life cycle. With this unique approach to warding off HIV infection, the enzyme produced by a person's own vulnerable cells could demolish the critical genetic instructions of HIV while not affecting his or her RNA.

"With this technique, we'd like to be able to offer an additional approach for patients who in their own
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Contact: Amy Adams
amyadams@stanford.edu
650-723-3900
Stanford University Medical Center
25-May-2004


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