Scientists have used the cutting-edge CRISPR/Cas9 gene-editing method to excise a key fragment of HIV’s genetic code from live rodents, Time Magazine reports. Publishing their findings in Gene Therapy, researchers employed a recombinant adeno-associated viral (rAAV) vector delivery system to send the gene-editing system into HIV-infected cells in live mice and rats that had been genetically engineered to have HIV incorporated into various types of cells throughout their bodies.
The gene-editing method was designed to cut a key fragment of HIV DNA from the genome of the animals’ cells.
The same researchers reported in March that they had successfully edited HIV DNA out of HIV-infected human cells in a laboratory setting. The paper led to a rash of erroneous media reports, stemming from an article in the United Kingdom’s The Guardian, claiming that a cure for HIV was only three years away.
Shortly after that paper was published, another research team put out a paper that found that HIV could become resistant to CRISPR/Cas9 gene editing, leading researchers to theorize that a multipronged gene-editing approach would be necessary to prevent such resistance.
The researchers in this new study injected the gene-editing apparatus into the rodents’ bloodstreams, and two weeks later analyzed the DNA in tissue samples from the animals. They found that the system had successfully snipped the targeted HIV fragment from more than half the cells of each type, including those in the tissues of the brain, heart, kidney, liver, lungs and spleen and in blood cells.
Analyzing the rats’ RNA, the investigators found that the gene editing had significantly lowered levels of viral RNA in circulating lymphocyte immune cells, as well as in the lymph nodes.
Update: A recent Temple University article on this study, which came out of the university, included the following note about the the paper’s authors:
“Editor’s Note: Drs. Khalili, Hu, and Kaminski are named inventors on patents that cover the viral gene editing technology that is the subject of this published journal article. In addition to the foregoing interests, Kamel Khalili, is a co-founder, board member, scientific advisor, and holds equity in Excision Biotherapeutics, a biotech start-up which has licensed the viral gene editing technology from Temple University for commercial development and clinical trials. Jennifer Gordon serves as an unpaid scientific advisor to Excision Biotherapeutics. The option to license the viral gene editing technology was signed prior to the acceptance of this manuscript. The authors declare that this work was produced solely by the authors and that no other individuals or entities influenced any aspects of the work including, but not limited to, the study conception and design; data acquisition, analysis and interpretation; and writing of the manuscript. The work was funded by the National Institutes of Health. No other entities provided funds for the work. The authors further declare that they have received no financial compensation from any other third parties for any aspects of the published work.”
To read the Time article, click here.
To read a press release about the study, click here.
To read the study abstract, click here.