Center for Gene Therapy Clinical Trials

Altering hematopoietic stem cells to fight HIV/AIDS
City of Hope researchers are currently working on two stem cell approaches to fight HIV/AIDS. One method, developed by John J. Rossi, Ph.D., Lidow Family Research Chair and chair of the Department of Molecular and Cellular Biology, is based on gene therapy with small ribonucleic acid molecules. These molecules block the genes HIV needs to infect T cells and will be used to modify the patient’s hematopoietic stem cells. The altered stem cells will then be infused into the patient, with the goal of “resetting” the immune system to produce T cells resistant to HIV infection. (COH Protocol #13282)

Another approach, developed by a team of investigators at City of Hope working with Sangamo Therapeutics and Keck School of Medicine of USC, uses an enzyme called a zinc-finger nuclease. This enzyme acts as a pair of molecular scissors that can edit a single gene in the HIV patient’s HSC that is needed by HIV for infection. The T cells resulting from the genetically modified stem cells will then lack this key protein the virus requires to infect cells. (COH Protocol #14017)