Trial for HIV Gene Therapy Begins

Following a theory that a particular gene could enable humans to become immune from the most common type of HIV, recruitment for gene therapy trials has began on February 3, 2009.  This experimental treatment targets HIV patients who have had problems with drug resistance.

After the quest to find a cure for HIV has become close to futile, as the drugs that have been tested has actually increased risk of HIV infection, researchers have shifted their focus to developing a therapy that would enable humans to become resistant to HIV.

"We do have good treatment for HIV.  That has been one of the most successful stories of the last 20 years in medicine," stated Pablo Tebas, an infectious disease expert at the University of Pennsylvania, in an interview with  "However, over time, if the medications are not taken properly, individuals develop resistance to the HIV treatments, so they tend to have more limited therapeutic options."

This comes after the discovery of a small number of people who were exposed to HIV yet did not get infected.  Scientists began working to discover the secret behind those people’s resistance (or possible immunity) against the virus, as well as how to make other people resistant to HIV.

They have discovered that most people have a gene called CCR5, making them vulnerable to HIV infections.  Those who have natural resistance towards the virus, however, have a mutant CCR5 that inhibits HIV.

Scientists have also found that by cutting the CCR5 gene out of white blood cells, which are involved in our body’s immune response known as T-cells, they could protect a test-tube full of human cells.  This gene editing technique relies on a type of proteins called zinc finger nucleases, which can delete any gene from a living cell.  In theory, zinc finger nucleases could provide that amount of immunity to anyone.

The experimental treatment involves taking some healthy T-cells from an HIV patient, and then clipping out their CCR5 genes.  Scientists then grow more of these edited T-cells in a dish before putting them back in the patient.

Tebas hopes that the results shown on the test-tube would also reflect in human experiments.

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