CRISPR-Based HIV Gene Therapy Administered To First Human Patient

In a clinical trial, the first patient has received a single dose of a new human immunodeficiency virus (HIV) gene editing therapy, researchers at the Lewis Katz School of Medicine at Temple University and Excision BioTherapeutics, Inc have reported.

In a collaborative effort, the researchers are currently running a phase 1/2 clinical trial to evaluate the safety and efficacy of their therapy, called EBT-101, which is based on gene editing technology known as CRISPR.

“Nearly 40 million people worldwide suffer from the effects of HIV, and more than 40 years after the discovery of HIV/AIDS, there still are no curative treatments,” said Professor Kamel Khalili, who helped lead the trial, in a statement.

“EBT-101 can potentially address long-standing unmet needs of individuals living with HIV/AIDS by removing viral DNA from their cells, thereby eradicating infection.”

When HIV infects, it takes long-term hold and hides from the immune system in cells, compromising the patient's immunity over time and eventually leading to the development of AIDS – the progressive failure of a patient's immune system. There is currently no cure for HIV, but various treatments and medications can help manage the infection and slow or prevent the progression of the disease.

The therapy currently being trialed, which uses CRISPR gene editing to remove HIV viral DNA from infected cells, is a major step toward finding a therapeutic cure.

Image source: Boghog / Wikimedia Commons.