An interdisciplinary group of researchers has claimed to have eliminated HIV from the mice genomes with the help of the gene editing tool CRISPR-Cas9 and a new drug. This is quite promising in the fight against HIV and AIDS, although a lot of work is still left before clinical trials can be started.
It may seem odd that a gene editing tool is used for removing infectious disease, however, HIV being a retrovirus embeds itself in DNA for replication. ART which stands for Antiretroviral therapy can help in suppressing HIV replication however it is not effective for eliminating the disease completely. The reason being it is incapable of purging cells where the virus has been dormant. The study has been published in the Nature Communications journal. When CRISPR-Cas9 is used with a newer form of ART, it eliminated the virus from the genome, a feat achieved for the first time.
Experiments were carried out on genetically modified mice which had similarities with human beings. The team led by Kamel Khalili, Lewis Katz School of Medicine, Temple University was successful in eliminating every single trace of HIV in about 30 percent of infected mice. Though not perfect, it provides hope to be optimistic. Khalili said that they can now proceed for trials in non-human primates, with clinical trials in humans within a year.
Khalili is also the founder and lead scientific advisor of Excision BioTherapeutics. This company uses CRISPR for treating viral diseases. It also possesses an exclusive license for the commercial application of the therapy. However, researchers have to proceed with caution as they have to prove that it is free of long term side effects such as cancer.
Antiretroviral therapy for the treatment of AIDS has benefitted many people all over the world, but it is not a cure technically. Patients have to be on a regular dosage of medicines for keeping the HIV virus in check. We are in a dire need of a therapy that eliminates HIV from the body completely.
Khalili took the help Howard Gendelman, professor of infectious diseases at the University of Nebraska Medical Center for the new study. He has been working on a new form of ART known as LASER. It stands for long-acting slow-effective release. It essentially targets the cells where HIV hides and suppresses its replication for long periods of time. For achieving this the drug is wrapped in nanocrystals, through which it spreads to tissues where HIV is supposedly dormant. It then slowly releases the drug.
LASER ART caught Khalili’s attention. It was incorporated with CRISPR-Cas9 and was successful in eliminating HIV from one-third of infected mice. Scientists have to proceed with caution as CRISPR has the risk of causing cancer in the body. Since it stays in the body for a long time it could cut other sites in an uncontrolled way, hence being cancerous.