This is possibly the biggest news about CRISPR so far. For the first time, researchers at Temple University have used CRISPR-Cas9 genome editing technology to cut HIV genes out of live animals infected with the virus. Is this the magic bullet that will lead to an effective treatment or a possible cure for HIV?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and is often used to refer to the CRISPR-Cas9 genome editing technology in which the CRISPR bacterial defense system is programmed to locate specific stretches of genetic code and edit DNA at these locations.
In simple terms, CRISPR acts like programmable molecular scissors that use an enzyme to make its cuts and slices. While this technique has been used in the past to cut HIV out of cells than have previously been removed from humans, a recent study published in Gene Therapy describes how this revolutionary technique has now been used successfully on live rats and mice.
In a proof-of-concept study, we show[ed] that our gene-editing technology can be effectively delivered to many organs of two small animal models and excise large fragments of viral DNA from the host cell genome.
That's a quote from Kamel Khalili of the Comprehensive NeuroAIDS Center at Temple University who led the team. They first engineered lab rodents having HIV genes in every cell in every organ of their bodies – brain, heart, kidney, liver, lungs and even blood. The team then used CRISPR molecular scissors cut out the HIV genes without harming the live rodents.
While not perfect, the CRISPR technique was successful in removing 50% of the HIV cells – still a monumental achievement. What even more exciting, the technique will be simple to apply to humans, says Khalili.
If this technology gets into the clinic to treat human patients, it’s not going to be very complicated. You don’t have to bring the patient to the clinic and do a bone marrow transplant or all kinds of complicated technology. You can basically apply this to any setting.
Is this a cure for HIV? Not yet, but it’s on the right path. Other HIV treatments do not eliminate HIV-1 from infected cells, allowing it to reactivate and replicate if treatment is ceased. Khalili is in the process of conducting a study on a larger group of animals and predicts there will be a clinical trial in the next few years.
Eradicating HIV is a noble and needed goal. Gene editing is revolutionary but controversial and begs the question, “What could possibly go wrong?”
Are we moving too fast? Will we be so enamored with progress that it becomes too late to stop a problem? The CRISPR horse is out of the barn. Only time will tell.