STEM CELLS SNIPPED OFF CCR5 RECEPTOR IF INFUSED IN HIV PATIENT MAY GROW NEW CELLS RESISTANT TO HIV /AIDS VIRUS

STEM CELLS SNIPPED OFF CCR5 RECEPTOR IF INFUSED IN HIV PATIENT MAY GROW NEW CELLS RESISTANT TO HIV /AIDS VIRUS
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Curing HIV, not simply controlling its replication, is the ultimate goal of HIV researchers. A new clinical trial at City of Hope could put that goal within reach.A novel treatment for HIV will be offered via clinical trial at City of Hope. The therapy edits a patient's stem cells to prevent production of a protein the virus needs to infect healthy cells.The trial will test an innovative new therapy that modifies the stem cells of patients with HIV to make them resistant to infection with the virus. Here's how it works:
The AIDS virus relies on a protein called CCR5 to penetrate and infect cells. For the trial, researchers will use a zinc finger nuclease, or ZFN, to modify blood stem cells from patients infected with HIV. The ZFN acts as a pair of molecular scissors that cuts the CCR5 gene out of the cells. Without the gene, there is no CCR protein – and researchers hope that when these cells are infused back into the patient, their immune system will be resistant to HIV.
“It’s erasing the gene, and once it’s erased, it never can reappear,” said John Zaia, M.D., in an interview with BuzzFeed News. Zaia is the Aaron D. Miller and Edith Miller Chair in Gene Therapy at City of Hope, chair of the Department of Virology and principal investigator of the trial.
He and other City of Hope researchers will work with the University of Southern California and Sangamo Biosciences, the biopharmaceutical company that developed the ZFN. The study is funded by the California Institute for Regenerative Medicine (CIRM) and was recently approved by the Food and Drug Administration.
“While we have a number of drugs that are effective in holding HIV at bay, we have nothing that cures it,” Zaia said. “In addition, for many patients, these medications come with significant long-term problems so there is a real need for a therapy that can help eradicate the virus from a patient completely. That is where our work is focused.”
— with Delhi Sacs and 19 others.
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