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Gene Therapy Studies

If you are interested in new approaches to possibly treat your HIV infection, the UCLA CARE Center's gene therapy studies may be an option for you.

Gene therapy involves removing some of your blood, changing some of the white blood cells (T-cells), and giving your blood back to you.


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Currently enrolling Cohort 5, this study is for HIV positive men and women ages 18 and older with an undetectable viral load for at least one year and CD4+ T cell counts ≥350 cells/mm3.  Participants will receive a single infusion of gene-modified CD4 T-cells at different doses depending on the enrollment group, followed by a structured interruption in HAART treatment, to see what effects the infusion has on possibly reducing HIV infection. 

What is the theory behind gene therapy and HIV?

Researchers are looking at a new way to possibly treat HIV called a "Zinc Finger Nuclease" (ZFN).  ZFNs are proteins that can modify another protein named CCR5.  This CCR5 protein is required for HIV to enter into and infect your T-cells.  The most important T-cells are those called "CD4+ T-cells," which express the CCR5 protein on the surface where HIV enters the cell.  By modifying CCR5 on CD4+ T-cells, the researchers conducting the study hope to show that HIV infection can be prevented in those cells. 

Some people are born without CCR5 on their CD4+ T-cells.  These people remain healthy and are resistant to infection with HIV.  Other people have a low number of CCR5 on their T-cells, and their HIV infection is less severe and is slower to develop into AIDS.  Therefore, researchers think that by modifying the CCR5 protein, they may be able to help your body to reduce HIV infection. 

How do "zinc finger" modified T-cells work?

Although you may have no detectable levels (or have intermittently very low levels) of HIV in your blood, HIV remains in some tissues in your body, primarily the gut tissue.  HIV infects the CD4+ T cells in your body, including the blood and gut.  Gene therapy studies will involve removing your white blood cells, which include CD4+ T cells.  The extracted CD4+ T cells are then genetically modified using ZFNs.  The genetically modified cells are then re-infused back into you.  Researchers hope that these genetically modified cells will be resistant to infection by HIV and will be able to reproduce additional resistant CD4+ T-cells in your body.  However, these studies are experimental, and this may not occur.

For more information about the UCLA CARE Center's gene therapy studies, please contact:
Phone:  (310) 557-9062
Email:  CAREOutreach@mednet.ucla.edu