New gene therapy could herald HIV breakthrough
Doctors have developed a new gene therapy that could control the HIV virus without the need for daily antiviral drugs.
A study from the biopharmaceutical company Sangamo BioSciences, published in the New England Journal of Medicine, used 12 patients and suggested the therapy was safe.
Researchers used genome editing to alter patients T-cells, which are part of the immune system, to replicate a rare mutation which protects only one per cent of the population from the HIV virus.
The altered cells were then multiplied and injected back into the patients. Out of the roughly 10bn cells that were injected back into patients, 20 per cent were successfully edited.
The therapy is designed to disrupt a gene, CCR5, used by HIV to infect T-cells. The mutation used by researchers to disrupt CCR5 is known as CCR5-delta-32.
Sangamo chief executive officer Edward Lanphier, said:
The target we are going after, CCR5, is the most advanced and most promising approach for a functional cure for HIV.
Four weeks after the infusion, half of the patients temporarily stopped taking their antiretroviral drugs so the researchers could examine the effect of the genome-editing treatment on the amount of the HIV virus in the patients’ bodies.
In four of these patients, the amount of HIV in the blood dropped. In one patient, the virus could no longer be detected at all. However, it was found that the best responder in the group already had one mutated copy of the CCR5 gene.