HIV Gene Therapy Trials Begin

Jim Burroway

February 3rd, 2009

Human trials are about to begin on a form of gene therapy that could immunize people against the most common type of HIV. Recruiting for the human trials began yesterday.

According to

Since the discovery that a small portion of people who are exposed to HIV do not get infected, scientists have been working to discover the secret to those people’s resistance and how to make others resistant as well.

It turns out that most people have a gene called CCR5, which makes them vulnerable to HIV infections. The naturally resistant people have mutant CCR5 genes that inhibit HIV.

Previously, scientists found that by cutting the CCR5 gene out of white blood cells involved in the immune response known as T-cells, they could protect a tube full of human cells from the virus. The gene editing technique relies on proteins called zinc finger nucleases that can delete any gene from a living cell.

Zinc finger nucleases are compounds that can slice open molecules. This one is is designed to go after the CCR5 gene. The treatment calls for removing CD4 T-cells,the immune cells affected by HIV, treating them with the drug, and re-infusing them into the patient. The hope is that these damaged cells will multiply and give the patient an immune system which is resistant to HIV.

The human trials are being conducted by Sangamo BioSciences, Inc., a California biotech company. The first phase is meant to look at safety and tolerably of a single infusion. The first people to receive the new treatment will be six patients who have developed drug-resistance to HIV and six other patients who are currently responsive to their existing drug regimen.

Suricou Raven

February 5th, 2009

An interesting idea, but this is making patients who have a mix of infectable and non-CCR5 T-cells. I’m not a biologist, but I know that HIV mutates and evolves extremally fast, and that this mix is the perfect environment for evolving a strain that can infect non-CCR5 cells too. I’d like to see some expert reassurance that this isn’t going to happen.

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