Daijiworld Media Network – Washington

Washington, Oct 6: In what could mark a historic leap in medical science, researchers are now closer than ever to developing a CRISPR-based therapy capable of cutting the HIV virus directly out of a patient’s genome — potentially paving the way for a permanent cure.

A US-based biotech company, Excision BioTherapeutics, has presented groundbreaking proof-of-concept data for its experimental CRISPR therapy, EBT-101, showing its ability to target and destroy dormant HIV reservoirs within the body.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a revolutionary gene-editing tool often described as “molecular scissors,” allows scientists to precisely cut and modify DNA. In this study, phase I and II clinical trials tested a novel CRISPR-Cas9 gene therapy as a potential functional cure for HIV infection.

Currently, HIV patients rely on Antiretroviral Therapy (ART), which keeps the virus dormant but does not eliminate it. The biggest challenge has been the latent reservoir of infected T-cells that harbor the virus’s genetic material, causing HIV to rebound once ART is discontinued.

The CRISPR-based approach, developed by Dr Kamel Khalili of Temple University and Nobel laureate Dr Jennifer Doudna of UC Berkeley, aims to overcome this challenge by directly editing out the viral DNA from infected cells.

In the trials, a one-time intravenous infusion of the therapy was found to be safe and well-tolerated, with no serious side effects. While most participants saw the virus return after stopping ART, one participant displayed a significant delay of nearly 16 weeks before viral rebound and a marked reduction in the size of the HIV reservoir.

Researchers say the results are early but promising — offering hope that, with higher-dose or refined delivery methods, CRISPR gene editing may one day achieve the world’s first true cure for HIV/AIDS.