"HIV Release" by Bette Korber at Los Alamos National Laboratory / Public Domain

Scientists use gene-edited stem cells to treat HIV — with mixed success

Sep 13, 2019

By Jonathan Lambert

For the first time, researchers have used CRISPR gene-editing technology to try to treat a person infected with HIV.

Scientists in China engineered human stem cells to mimic a rare form of natural immunity to the virus and transplanted them into a man with HIV and blood cancer. The gene-edited cells survived in the man’s body for more than a year without causing detectable side effects, but the number of cells was not high enough to significantly reduce the amount of HIV in his blood.

“This is an important step towards using gene editing to treat human disease,” says Fyodor Urnov, a biologist at the University of California, Berkeley. “Because of this study, we now know that these edited cells can survive in a patient, and they will stay there.”

The study1 was published on 11 September in The New England Journal of Medicine. Lead author Hongkui Deng, a biologist at Peking University in Beijing, says that the research was inspired by a remarkable bone-marrow transplant that seemingly cured a man of HIV more than a decade ago.

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