Researchers from Temple University School of Medicine have discovered how to permanently extricate HIV-1 from human cells, possibly avoiding the need for lifelong drug treatment. Though in its very early stages, this may prove to be a critical step in permanently defeating the disease.
At the closing ceremony of the AIDS 2014 conference last week in Melbourne, Australia, many of the speakers, including longtime AIDS researcher and International AIDS Society Presidential Award winner Eric Goosby, told of how utterly terrifying the disease seemed 30 years ago. That fear has not left. However as the medical community and wider community has learned more about the disease, the resolution to fight it – and destroy it by 2030 according to UNAIDS – has only become stronger. Hope now sits beside abject fear. Temple University’s new discovery may yet be cause for greater hope. One of the main issues in the treatment of HIV-1 is not simply that it is expensive, but that antiretroviral therapy can only stay the illness, not destroy it. Many ARVs also have terrible side effects and they can speed up diseases more commonly associated with aging. They also may cause problems related to co-infections, such as Hepatitis C, where liver degradation is sped up by antiretroviral treatments.
Added to this is that HIV is a tricky and tenacious disease: it becomes part of a patient’s DNA making it nigh impossible to eradicate.
However researchers from Temple University School of Medicine have found a way to cut the infected genes out, potentially eradicating the virus for good and negating the need for lifelong ARV treatment. Dr Kamel Khalili, Professor and Chair of the Department of Neuroscience at Temple calls it an “important step” towards the eradication of AIDS, though it is still years away from the clinical stage.
The technique uses a DNA-snipping enzyme, a nuclease, and a targeting RNA strand to hunt down the genome and cuts the HIV-1 DNA from it. The cell is able to repair its own genomes, essentially sewing itself together again, only now HIV-free.
This treatment will work in varied cell types such as the T-cells and monocytic cells that harbor HIV. In designing the molecular tools, researchers chose nucleotide sequences that do not appear in any coding sequences of human DNA to avoid what they call off-target effects, where patient’s cells or own DNA might be damaged.
The technique may also be applicable against many other viruses.
There are still serious hurdles, though – how to get the treatment into each, individual cell being the main one. Also, HIV-1 is known for mutations and there can be no single, prescriptive treatment for it. Treatments may need to be somewhat bespoke as every patient has their own viral sequence. However another potential upside is that there is the chance this may be used not simply as a treatment but also a vaccine as cells containing the nuclease-RNA combination do not acquire the HIV infection.
"We want to eradicate every single copy of HIV-1 from the patient," said Dr Khalilli. "That will cure AIDS. I think this technology is the way we can do it."
Dr. Khalili discusses the findings in the Temple University video below.
Source: Temple Health