Will Gene Editing Help Rid The World of HIV


With the progress that has followed with antiretroviral therapy suppressing HIV, the virus that causes AIDS, many patients are left with latent viral “reservoirs” that hide in the body and can resurge at any time. Two recent advances are focused on using new technology to combat these HIV reservoirs.

Last week, scientists at Case Western Reserve University received an $11 million grant from the National Institutes of Health to study gene-edited T cells in HIV. The Case Western team is working with Sangamo Therapeutics to design a study in 20 patients, which they anticipate will begin this year, according to a press release.

The probability of taking infection-fighting T cells from patients and directing them to recognize and eradicate illnesses from the body has already been proven in oncology with two FDA-approved blood cancer treatments: Novartis’ Kymriah and Gilead’s Yescarta. While those treatments are designed to train T cells to recognize and target an antigen on cancer cells, the Case Western-developed technology works by disrupting HIV’s ability to enter cells.

The technology is centered on eliminating a gene called CCR5, which is responsible for ushering HIV into host cells. The technique, called zinc finger nuclease gene editing, employs specially engineered proteins to create precise breaks in DNA, knocking out CCR5.

Sangamo had shown in earlier trials that T cells edited in this manner can be expanded outside the body and that they persist and grow after being infused back into patients. They also documented increases in CD4+ T cells, which helped break down the HIV reservoirs. During the new trial, 10 patients will receive gene-edited T cells and the other 10 will be given unmodified T cells, according to the statement.

“For patients with HIV, new treatments to permanently eradicate latent HIV reservoir and increase CD4+ T cell counts—potentially leading to a cure—are an important unmet need,” said Rafick-Pierre Sekaly, Ph.D., a professor of immunopathogenesis at Case Western Reserve School of Medicine, in the statement.


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