BALTIMORE — An ambitious effort to cure HIV with CRISPR genome editing fell short in an early clinical trial, investigators announced Friday morning.
In the study, run by Excision BioTherapeutics, researchers tried to use the gene editing tool to address a chief reason HIV has been so hard to cure. While antiviral drugs can clear patients of replicating virus, HIV is able to worm its way into a patients’ own DNA in certain cells. If the patient ever stops taking medicines, those cells start pumping out HIV particles and the infection roars back.
Researchers hoped they could send CRISPR to those cells and, by cutting the HIV DNA lurking there at two spots, slice out the virus. In the Phase 1 trial, investigators administered the treatment to five patients. They then took three of them off conventional antiviral treatment.