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Gene Therapy

Apr 18, 2006

Hi Dr, Bob

What do you think about gene therapy ? The San Francisco Chronicle reported that Gene therapy has promissing result I just like to hear from an expert like you thanks Doc


Response from Dr. Frascino


I assume you are referring to the gene therapy clinical trial currently underway in San Francisco, Los Angeles and Sydney. For our readers unfamiliar with this study, I'll briefly explain. The 74 clinical trial participants first spend 16 hours hooked up to a machine that filters out stem cells and returns the rest of the blood to the body. This is called aphoresis. Next, the harvested stem cells are infected in the laboratory with a harmless mouse virus specifically engineered to carry the ribozyme gene. These genes are genetic blueprints that tell the cell how to make the enzyme ribozyme. Ribozyme is a custom-built chemical "scissors" that cuts up one of HIV's nine genes just as the virus tries to replicate itself. Three days after the gene is transferred into the stem cells, these genetically altered stem cells are returned to the patient's bloodstream. In subsequent weeks, these gene-modified stem cells produce a variety of infection-fighting white blood cells, each containing the ribozyme that we hope will be able to ward off HIV. If all goes according to plan, when HIV tries to infect these fortified blood cells, the virus will be destroyed. Ultimately we may even be able to rebuild or reconstitute the HIV-damaged immune system.

As you can see, this is very high-tech stuff. One of my good friends, Dr. Ron Mitsuyasu, director of the UCLA Center for Clinical AIDS Research and primary investigator on this clinical trial in Los Angeles, collaborated with the Australian team on the first pilot study of the new therapy, showing it was safe. None of us, including Dr. Mitsuyasu, believes this complex therapy will replace effective antiretroviral drugs. However, studies like these are opening new avenues of research that allow us to explore ways of using the immune system, gene therapy and stem-cell therapy in combination to work towards our ultimate goal a cure for HIV.

Stay tuned to this site, Tony. We'll keep you updated as this and other immune-based-therapy stories evolve.

Dr. Bob

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