The first experimentation to eliminate HIV with genetic editing has started

Clinical trials of EBT-101, a treatment that uses Crispr-Cas9 technology and could represent the first therapy to eradicate HIV infection from human cells, have been authorized in the United States

(photo: Callista Images / Getty Images) Eliminate HIV directly from human cells through Nobel Prize Crispr-Cas9 technology. Thinking about it a handful of years ago, when the first in vitro and then animal studies appeared in scientific journals, it seems almost unbelievable. Instead, a potential therapy that could definitively cure HIV infection exists and is called EBT-101. The Food and Drug Administration (FDA), the US regulatory body, has just authorized phase 1 and 2 clinical trials, aimed at verifying the safety and efficacy of this treatment, developed by Kamel Khalili and his team of researchers of Temple University, in the United States, and the biotech company Excision BioTherapeutics, with the collaboration of a team of researchers from the State University of Milan.

Eliminating HIV definitively

Since HIV, the human immunodeficiency virus, was first identified at the beginning of the 1980s, therapies against viral infection have undergone considerable progress, thanks above all to the development of antiretroviral drugs, to date the only ones available to contain the viral infection. It is a class of drugs targeted against different components of the virus, but which have the common purpose of blocking the molecular machine responsible for HIV replication.

Thanks to these molecules, progressively improved over the years, not only has it been possible to obtain a reduction in the reproduction of the virus in the cells, but also a significant decrease in the viral load in the blood and consequently an improvement in functions of the immune system.

Yet, antiretroviral therapy does not allow to eliminate the virus permanently - and therefore to cure the infection in the sense of the word - forcing patients to take these drugs for life and , in case of interruption of therapy, generating a surge in viral load in the weeks immediately following (called, in technical jargon, viral rebound). In addition, people with HIV undergoing antiretroviral therapy often have symptoms associated with the side effects of the treatments and the latent presence of the virus inside the cells.

A seven-year process

For these reasons, seven years ago, Khalili's research group, in collaboration with the University of Berkeley team of future Nobel Prize winner Jennifer Doudna, began experimenting with a new therapy involving the use of the technique of Crispr-Cas9 gene editing to recognize and eliminate the proviral HIV DNA (i.e. the portion of the virus that integrates within the host cell) and consequently inactivates its expression, and not just its replication, as happens with antiretroviral therapies.

“An exciting development”

The researchers started from studies on human cells in vitro, and then experimented the technique underlying EBT-101 on different animal models i, including primates. The developed treatment uses an adeno-associated virus (a type of viral vector capable of carrying portions of genetic material inside cells, normally also used in other gene therapies) which allows to provide the cells of the immune system with a one-off treatment. and permanent against HIV.

Thanks to the vector, in fact, the components of Crispr-Cas that eliminate the proviral DNA are inserted inside the cells: in preclinical studies, the treatment was able to successfully cure HIV infections . Now the experimental program authorized by the FDA employs, in a way very similar to what has already been verified on animal models, Crispr-cas9 and two rna molecules that target three sites within the HIV genome, thus removing large portions of the virus. and minimizing potential mechanisms of viral escape.

The group of Pasquale Ferrante of the State University of Milan participates in the project, both in the preclinical phase and in these subsequent phases. "The ability to test this treatment in people living with HIV is an exciting development, and it is certainly of interest to millions of people," comments the researcher.


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