An HIV-positive man in China is doing well after receiving infusions of donor cells whose genes were edited with CRISPR 19 months ago, scientists reported on Wednesday in the New England Journal of Medicine. That makes him the longest-followed individual ever to be treated with CRISPR, a genetic technology that has inspired sky-high hopes for disease cures and spawned a multibillion-industry in just four years.
The patient’s mere survival, apparently without side effects from a treatment that a few studies suggested might trigger cancer or other disastrous genetic damage, provides some assurance that CRISPR-based therapies might be safe — and overshadows the fact that the intervention fell well short of its goal: eliminating HIV from the man’s cells.
“They attempted a moonshot, and while they did not land on the moon, they got back home safely,” said Fyodor Urnov of the Innovative Genomics Institute at the University of California, Berkeley, who previously helped lead a study for Sangamo Therapeutics (SGMO) of whether an older genetic technology, zinc fingers, could cure HIV/AIDS. Crucially, Urnov said of the Chinese study, “they highlighted... see more