In what’s being called a major step to a cure for HIV, researchers at the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) have for the first time succeeded in completely eradicating the HIV virus from the genomes of a living animal.
“Our study shows that treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals,” said Kamel Khalili, PhD.
The study, published on Tuesday July 2 in the journal Nature Communications, used mice engineered to produce human T cells that are susceptible to infection. The mice were treated with a combination of gene editing and gene therapy known as CRISPR-Cas9 and LASER ART after HIV infection was established.
Antiretroviral therapy (ART) is the current form of HIV treatment that suppresses HIV replication but does not eliminate the virus from the body and it requires life-long use. If someone with HIV stopped taking ART treatment, the HIV rebounds and begins fuelling the development of AIDS because the virus
is able to integrate its DNA sequence into the genomes of cells of the immune system, lying dormant.
Long-acting slow-effective release or LASER ART was packaged into nanocrystals that readily distribute to tissues where HIV is likely to be lying dormant. From there, they are stored within cells for weeks and slowly release the drug.
At the end of the treatment period analyses on the mice revealed the complete elimination of HIV DNA in about one-third of HIV-infected.
“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 non-human primates and possibly clinical trials in human patients within the year”.