Scientists have successfully eliminated the HIV genome from human cells, a breakthrough that could potentially pave the way for a cure for HIV/AIDS.
A research team from the NeuroAIDS Center at Lewis Katz School of Medicine – Temple University, Philadelphia, used a new DNA editing technique to remove the HIV genome from CD4+T infected cells.
Using the CRISPR/Cas9 gene editing technique, the scientists managed to eliminate HIV-1 DNA from T cell genomes in lab experiments, and prevented reinfection after the cells were re-exposed to the virus.
Following the removal of the HIV genome, the edited cells continued to function normally, with no signs of damage, and appeared to be protected from reinfection.
The CRISPR/Cas9 gene editing method uses RNA proteins to alter targeted sections of DNA in a cell. It involves using naturally occurring enzymes which form part of a bacteria’s immune system to target viral DNA.
The team at Temple had previously edited HIV DNA out of human cell lines. The new study, used patient cells grown in the lab, showed unequivocally that cleared cells were no longer susceptible to infection by HIV.
Dr. Kamel Khalili, Chair of Temple University’s Department of Neuroscience and Director of the Center for Neurovirology at Temple University’s Lewis Katz School of Medicine, said that “the findings are important on multiple levels.” In a press release he explained that the results “demonstrate the effectiveness of our gene editing system in eliminating HIV from the DNA of CD4 T-cells and, by introducing mutations into the viral genome, permanently inactivating its replication.
“Further, they show that the system can protect cells from reinfection and that the technology is safe for the cells, with no toxic effects.”
Using T-cells from HIV patients grown in cell culture, the scientists reported using the gene editing method lowered viral load in the patient’s cells, suggesting it could be used as a treatment.
“These experiments had not been performed previously to this extent,” Khalili said “But the questions they address are critical, and the results allow us to move ahead with this technology.”
Encouragingly, the technique may be successful at eliminating HIV-1 virus from hidden reservoirs which have proved previously an obstacle to potential cures.
“The results point toward this approach as a promising potential therapeutic avenue to eradicating HIV-1 from T reservoir cells of host patients, to prevent AIDS re-emergence,” claimed the research team in their Nature article.
The new genome editing technique could potentially lead to a cure of HIV/AIDS and furthermore revolutionise the way genetic diseases are treated.
Dr. Thomas Malcolm, of Excision BioTherapeutics Inc., a gene editing company that holds exclusive rights to commercialise the technology, believes this is a critical step in progressing the technology towards human trials. “This is a major advance in safety and efficacy for the use of CRISPR/Cas9 gRNA HIV eradication for use in humans,” says Dr. Malcolm. “These exciting results also reflect our ability to select viral gene targets for safe eradication of any viral genome in our current pipeline of gene editing therapeutics.”