By Charles Casey
An improved gene therapy strategy using modified human stem cells shows promise in animal models as a functional cure for HIV.
The achievement, which involves an improved technique to purify populations of HIV-resistant stem cells, opens the door for human clinical trials that were recently approved by the US Food and Drug Administration.
“We have devised a gene therapy strategy to generate an HIV-resistant immune system in patients,” says Joseph Anderson, principal investigator of the study and assistant professor of internal medicine at University of California, Davis.
“We are now poised to evaluate the effectiveness of this therapy in human clinical trials.”
Anderson and his colleagues modified human stem cells with genes that resist HIV infection and then transplanted a near-purified population of these cells into immunodeficient mice. The mice subsequently resisted HIV infection, maintaining signs of a healthy immune system.
The findings are now online and will be published in the journal Stem Cells.
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