CRISPR Gene Editing and Long-Acting ART: A Dual Therapy Strategy for HIV Elimination in Mice

Shortly after the human body is infected with AIDS virus (HIV), the DNA of the virus will be embedded in the genome of the host.Antiretroviral therapy (ART)The main reason why there is no cure for AIDS, despiteART therapy has extended the life expectancy of AIDS patients to that of uninfected people, but patients must take drugs every day to suppress the virus and may also lead to drug resistance or its complicationsTherefore, it is still necessary to find a better treatment method in clinical practice.

U.S.Temple UniversityandUniversity of Nebraska Medical Center (UNMC)In a new study published in the Proceedings of the National Academy of Sciences of the United States on May 1 this year, the research team pointed out that they used humanized mice infected with HIVCRIPR gene editing technique inactivates two genesand combinelong-acting antiretroviral therapytreatment, it was found thatSuccessfully eliminated HIV virus in 60% of micecompared with the 29% of the experimental data first published in 2019, there is a considerable progress.

In the new study, the researchers first injected HIV-positive mice withLong-acting slow-effective release antiretroviral drugs (long-acting slow-effective release ART, LASER-ART)use it after one monthCRISPR-Cas9 gene editing technique inactivates CCR5 genewhen the expression level of CCR5 gene dropped to the lowest after seven days, the second CRISPR-Cas9 treatment was further initiated toRemoval of HIV-1 DNA embedded in the host genome。

In order to ensure that the HIV virus will not rebound, the researchers continued to use a variety of highly sensitive techniques to track the virus in the mice for the next two months. In addition to analyzing the blood, they also checked all organs known to be infected by the virus. The experimental results showed thatSix out of 10 mice in the treatment group were completely free of HIVsimilar results were obtained in the second round of repeated studies,5 of 9 treated mice had undetectable viral load。

CRISPR technology is still the preferred method of eliminating the HIV virus, and the use of CRISPR to inactivate CCR5 to prevent the virus from spreading, the researchers said.Dual therapy strategy promises to improve treatment outcomes and permanently eliminate virus。

For those mice where the virus still rebounded, the researchers attributed the possible cause todrug delivery systemcapabilities, will also be committed to developing in the futurehighly targeted nanoparticlesAnd otherModified and highly efficient, harmless viral vectorsthese vectors can bind to the CCR5 receptor and deliver HIV-1 gene therapy directly to the active site of the virus,The team is currently conducting phase I/II clinical trials of HIV-1 gene therapy, with the ultimate goal of bringing this therapy into the clinical treatment of AIDS.

Source: Fierce Biotech, National Academy of Science

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