UNMC announces HIV research breakthrough

Henrietta Brewer
July 3, 2019

In rats and mice, they showed that the gene editing system could effectively excise large fragments of HIV DNA from infected cells, significantly impacting viral gene expression.

Using a gene-editing tool like CRISPR to clear out an infectious disease may seem unusual, but HIV is a retrovirus that embeds itself within DNA as a means to replicate.

That's correct, completely and successfully eliminated HIV from an infected animal.

It marks a critical step towards a possible cure for humans.

Researchers discovered a way to create a cure for HIV. "That's one of the challenges right now companies are dealing with: How do we fund al this mandated pre-clinical and clinical work and still have and still be able to recoup some profit on the backside?" CRISPR-Cas9 is a gene editing tool that's been hailed as a breakthrough technology, as it has the potential of curing genetic diseases.


"This achievement could not have been possible without an extraordinary team effort that included virologists, immunologists, molecular biologists, pharmacologists, and pharmaceutical experts", said Howard Gendelman, M.D., Margaret R. Larson Professor of Infectious Diseases and Internal Medicine, chair of the UNMC Department of Pharmacology and Experimental Neuroscience and director of the Center for Neurodegenerative Diseases. According to him, the number of cells with HIV that must be destroyed in a human body is much higher. Patients have to take medicines on a regular basis to keep the HIV virus in check, lest it re-emerge in the body and proliferate to unsafe levels.

The virus is at present treated with antiretroviral therapy (ART), which suppresses it from replicating and prevents many patients within the US from developing AIDS.

Researchers used a new form of ART called LASER ART on 23 "humanized mice", animals genetically modified to bear similarities to the human immune response.

It combines this with a recently developed therapeutic strategy known as LASER (long-acting slow-effective release) ART. This is of particular use in this instance as the antiretroviral drug can be stored in nanocrystals, slowly releasing the drug where the virus is found.

This permitted long-term viral infection and ART-induced latency. In one-third of the sample, researchers only involved LASER ART alone, while another third used CRISPR-Cas9.


"While ART has transformed HIV-1 infection into a chronic treatable disease, virus persists in tissues that include the gut, lymph nodes, brain, spleen amongst other sites".

At the end of the treatment period, mice were examined.

An worldwide team of virologists were able to destroy all trace of HIV in the cells of the two "humanized" mice with the genomic editor CRISPR/Cas9.

"The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection", Dr Khalili said.

"We now have a clear path to move ahead to trials in nonhuman primates and possibly clinical trials in human patients within the year", added Khalili, who also directs Temple's neurovirology center and its neuroAIDS center.


Earlier this year health secretary Matt Hancock promised by 2030 there would be no new cases of HIV in England.

Other reports by iNewsToday

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