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UC Davis researchers exploring gene therapy to fight AIDS
Date:12/5/2008

lish similar immunity in HIV patients, the UC Davis team manipulated human skin cells to give these cells many of the same properties as stem cells. These transformed cells, called induced pluripotent stem (IPS) cells, are capable of differentiating into, among other cell types, hematopoietic stem cells, which are normally found in bone marrow and are responsible for producing the various types of immune cells.

"If we can replace normal immune cells with HIV-resistant ones, we can cure AIDS," Bauer said.

Bauer and stem cell program research associate Joseph Anderson have developed several anti-HIV genes that they plan to insert into IPS cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). These engineered IPS cells could then be differentiated into bone marrow stem cells and introduced into the patient using a procedure similar to a bone marrow transplant.

"The hope is that one day we will use a patient's own skin cells to develop the engineered IPS cells to avoid possible rejection," said Bauer, who worked on clinical HIV gene therapy trials at Childrens Hospital Los Angeles before coming to UC Davis. "As in the German case, the end result would be an immune system that produces HIV-resistant immune cells."

In theory, the experimental treatment would result in an immune system that remains functional, even in the face of an HIV infection, but would halt or slow the progression toward AIDS.

"The anti-HIV genes take advantage of how HIV works," added Anderson, who is now writing a paper about the investigation. "The virus targets cells that are descendants of hemopoeitic stem cells."

During the first stages of infection, HIV targets macrophage cells, gaining entrance into the cell by binding to a receptor called CCR5 on the cell's surface. Later in the infection it targets CD4+ T cells, binding to the CXCR4 receptor on the surface of
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Contact: Charles Casey
charles.casey@ucdmc.ucdavis.edu
916-734-9048
University of California - Davis - Health System
Source:Eurekalert

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