Researchers Successfully Eliminate HIV in Mice With the Help of CRISPR

New technologies have been able to successfully eliminate the HIV virus in mice, report researchers from Temple University and the University of Nebraska Medical Center. The combination of CRISPR, a gene-editing tool, and LASER ART, long-acting slow-effective release (LASER) and antiretroviral therapy (ART) has eliminated HIV DNA in the genomes of mice according to a study published in the journal Nature Communications.2

CRISPR in HIV research

This is a new therapeutic strategy that combines the ongoing research efforts of two independent groups who realized they could combine their technologies to effectively eliminate replication-competent HIV DNA (infection capable of replicating itself) from genomes.1,2 The dual strategy was able to remove the virus in nearly one-third of humanized mice that were infected with virus.1,3 Humanized means the mice were injected with human bone marrow which mimics our immune system.2

Although HIV continues to be an incurable condition, antiretroviral therapy (ART) is able to virally suppress the HIV infection. This means it can remain in a latent, non-replicating state.1 But if a person stops taking antiviral medications, the virus will reactivate and the disease is able to progress, possibly to the late-stage HIV infection (AIDS).1,2

CRISPR gene editing

CRISPR-Cas9 is a gene-editing tool.2 Scientists can use this technique to change DNA by adding, removing or changing genetic material. LASER ART is a “super” form of ART; it keeps the replication of the virus at low levels for longer periods of time.2

Why was LASER ART used?

The LASER ART approach improves the ability of CRISPR to genetically edit out, effectively cleanout, or remove the viral DNA.1-3 It does this by suppressing the virus’s ability to replicate.1 Antiretroviral medications are then stored in nanocrystals, which allow for an extended, slow release of the drugs at the source of the virus.2 Researchers targeted tissue in the spleen, bone marrow, and brain where inactive HIV cells often hide.3

How did researchers use CRISPR and LASER ART?

How did they apply this therapeutic approach? Researchers first used LASER ART to impede the growth of, and decrease, the amount of HIV. This was followed by CRISPR treatment which study authors describe as “chemical scissors” which were able to “eliminate the residual integrated HIV DNA still present.”2

How will CRISPR impact future research?

Lead investigators suggest that these advancements will pave the way for future trials, first in non-human primates which are presently underway, and eventually in human clinical trials.1,2 With FDA approval, the hope would be to begin human trials at some time in 2020.3 The authors recognize that what is effective in mice may not work as well in humans.2 The size of humans can make the administration of the treatment and quantity of DNA needing to be destroyed a challenge.

The hope is that this research will lead to a cure for HIV in humans. Almost 38 million people are living with HIV, 1.2 million of whom are in the United States.2,3

HIV has been successfully managed with ART, and requires lifelong use to control and suppress the HIV viral load in persons living with the chronic condition.3 These new research findings on CRISPR may be an important step toward the goal of finding a cure for HIV.3

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