- Researchers are working to suppress HIV infection using genome-editing technologies.
- They seek to make HIV-resistant cells by disrupting the gene that encodes the CCR5 coreceptor.
- 8 patients are currently in treatment in this trial.
Why this matters
- Genome-editing therapies could control HIV infection through cells engineered for infection resistance.
- Researchers are working to isolate human stem cells and engineer them to be CCR5-negative.
- Their goal is drug-free control of virus replication or eradication by developing therapies that become a 1-time treatment.
- Extracted stem cells from patients are treated with busulfan to allow for their return after engineering.
- At 9 months, patients who have CCR5-negative edited cells in the blood may undergo an analytical treatment interruption (ATI) and start taking antiretroviral drugs to see if the treatment has had any effect.
- If HIV starts to come back, the researchers expect that the edited cells will have a selective advantage in controlling the virus.
- 14 patients are enrolled, 8 have been treated, and 2 of those have gone long enough that they have reached the ATI stage.
- The planned follow-up period is 15 years.