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

these cells and bringing on full-blown AIDS.

What researchers discovered is that there is a natural mutation in less than 1 percent of Caucasians that results in a lack of CCR5 receptors on any of their cells.

"We also found that these people are naturally resistant to HIV," said Bauer. "So, more than 10 years ago, we began our work creating a gene that would knock down expression of CCR5 and other key receptors and interfere with other routes of HIV infection."

For IPS-based anti-HIV gene therapy to become reality, UC Davis researchers must first conduct safety and efficacy trials. Researchers have created a mouse model that replicates a normally functioning human immune system.

"We can now move forward and test the safety of the viral vectors, as well as the ability of anti-HIV genes to inhibit HIV infection," noted Anderson. "The humanized mouse model is an important step toward bringing this possible cure to patients."

Bauer and Anderson are hoping to demonstrate in their mouse model that HIV-infection cannot occur following their gene therapy treatment, providing the needed confidence in safety before embarking on clinical trials. This work and studies on the clinical use of IPS cells, Bauer predicts, will lead to a cure for AIDS.

"A real cure will come when we can replace all the hematopoietic stem cells with HIV resistant stem cells. What is so exciting is that we're clearly on the path of doing just that," said Bauer.


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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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