Gene therapy to fight HIV studied at US university

Gene therapy to fight HIV studied at US university

PHILADELPHIA (Ivanhoe Newswire/WTVM) - For the first time ever, doctors at the University of Pennsylvania have successfully used personalized gene therapy in a dozen patients with HIV, knocking the virus down to the point where it was almost completely undetectable.

Researchers say it's a huge win in the fight against aids and HIV, and brings them closer than ever to a cure.

Researchers have found a way to control HIV without the use of drugs by doctoring a patient's cells to resist infection. The target is one protein on the surface of white blood cells called a CCR-5 receptor.

"People without CCR-5, they are resistant to HIV, so what we do, is we try to take patients who have HIV infection and remove the CCR-5," Pablo Tebas, M.D., Infectious Disease Specialist at the University of Pennsylvania says.

Penn researchers have taken white blood cells from patients and modified them with a specially designed molecule called a zinc finger nuclease. It causes mutation that reduces CCR-5 on the surfaces of the cells; without it, the HIV cannot enter.

"This is like a cruise missile specifically directed to this one gene in all of the 23-thousand genes in the human genome," Bruce Levine, PhD., Pathologist at the University of Pennsylvania says.

The modified cells are reproduced in the lab without the CCR-5, frozen, and then infused back into the patient.

"The cells are grown for ten days, the testing takes a few more days, so we can go what we call vein to vein in about two to three weeks," says Dr. Levine.

In most of the patients, doctors noted a dramatic spike in the modified cells one week after the infusion. They also detected modified cells in lymph tissue, suggesting the cells were functioning normally.  Researchers say this is not a cure for HIV, but one step in a combination that may someday make the virus obsolete.

Researchers say some of the patients who went off their normal anti-retroviral drugs completely during the gene therapy retained lower amounts of the HIV in their systems- including one patient whose HIV levels became undetectable.

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