Tuesday, July 14, 2015

Stem cell gene therapy for eliminating HIV infection

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research are one step closer to engineering a tool that could one day arm the body's immune system to fight HIV, and win. The new technique harnesses the regenerative capacity of stem cells to generate an immune response to the virus.
"We hope this approach could one day allow HIV-positive individuals to reduce or even stop their current HIV drug regimen and clear the virus from the body altogether," said Scott Kitchen, the study's lead author and a member of the Broad Stem Cell Research Center. "We also think this approach could possibly be extended to other diseases." Kitchen also is a member of the UCLA AIDS Institute and an associate professor of medicine in the division of hematology and oncology at the David Geffen School of Medicine at UCLA.Kitchen and his colleagues were the first to report the use of an engineered molecule called a chimeric antigen receptor, or CAR, in blood-forming stem cells. Blood-forming stem cells are capable of turning into any type of blood cell, including T cells, the white blood cells that are central to the immune system. In a healthy immune system, T cells can usually rid the body of viral or bacterial infection. But HIV is too strong and mutates too rapidly for T cells to fight against the virus.The researchers inserted a gene for a CAR into blood-forming stem cells in the lab. The CAR, which is a two-part receptor that recognizes an antigen, was engineered to be carried by T cells and direct them to locate and kill HIV-infected cells. The CAR-modified blood stem cells were then transplanted into HIV-infected mice that had been genetically engineered with human immune systems. (As a result, HIV infection causes disease similar to that in humans.)
The researchers found that the CAR-carrying blood stem cells successfully turned into functional T cells that could kill HIV-infected cells in the mice. The result was a decrease in HIV levels of 80 to 95 percent.

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