Scientists Eliminate HIV in Live Animals for the First Time Using CRISPR Gene Editing Technique

There may be a lack of a permanent cure for HIV infection but scientists might be inching nearer to finding a solution. Temple health researchers managed to eliminate HIV from live animals fot the first time using gene CRISPR editing.

The study was published on May 3 in the journal Molecular Therapy. Researchers from Lewis Katz School of Medicine at Temple University (LKSOM) and the University of Pittsburgh conducted the study, which shows that they can successfully eliminate HIV DNA from animals.

"Our new study is more comprehensive," Dr. Wenhui Hu, associate professor in the Center of Neuroscience at LKSOM, said in a statement. "We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy."

Researchers perform the process in three animal models. Some were even "humanized" using mice with infused human immune cells. The process worked for all living animal models, eliminating HIV DNA successfully. This proves that HIV-1 can be contained and eliminated by gene editing technology known as CRISPR/Cas9. This new gene editing technique was derived from trial and error and by improving previous studies on HIV cure.

"During acute infection, HIV actively replicates," explained co-investigator Kamel Khalili, Ph.D., professor and chair of the department of neuroscience at LKSOM, said. "With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection."

In the new process, the team used EcoHIV strain to infect the mice -- a type of HIV similar to human HIV-1. They found out that during the infection process, HIV replicates. With the new process using CRISPR/Cas9, they managed to contain or block the proliferation of HIV. Based on the study, the success of the experiment is at 96 percent using EchoHIV. Being similar to the human HIV, this is the first time HIV-1 was eradicated by CRISPR/Cas9 system or an advance prophylactic treatment.

To kill the virus, the team used "adeno-associated" viral (rAAV) vector based on subtype AAV-DJ/8. They improved the gene editing apparatus to contain more RNAs to help eliminate HIV. The researchers believe that this is the biggest accomplishement on HIV resarch. However, it is yet too far to determine a formal HIV cure.