Chinese scientists develop targeted gene-editing system aiming for new HIV cure
The new therapy uses exosomes to carry Cas12a into cells to accurately locate the HIV virus, including latent HIV, and shreds its genome, achieving a functional cure for AIDS. PHOTO: PEXELS
A research team at the Wuhan University of Science and Technology (WUST) has recently developed a new targeted delivery system to send gene editing tools into cells. The tools will precisely locate the HIV virus and cut its genome into fragments, thereby achieving a functional cure, the China Science Daily reported on Tuesday.
Current HIV treatment methods include the widely used cocktail therapy, as well as emerging approaches like immune cell therapy and gene therapy. Cocktail therapy aims to suppress viral replication to the greatest extent, improving the quality of life and survival rates of patients. However, it cannot eradicate the virus.
Immune cell therapy can only identify and eliminate cells that are actively replicating the virus, leaving dormant infected cells unchanged. Existing gene therapies, such as those using adeno-associated viruses as delivery vectors, suffer from shortcomings such as poor targeting capabilities and toxicity due to excessive dosages.
For the WUST study, the results of which have been published in the Molecular Therapy journal, a team led by Gu Chaojiang invented the exosome-mediated targeted CRISPR-Cas12a delivery system (EMT-Cas12a) based on engineered exosomes.
Exosomes are tiny vesicles secreted by cells, carrying biological information such as proteins and RNA, and they act as couriers between cells. Cas12a -- often referred to as "gene scissors" -- is a gene-editing tool capable of cutting DNA precisely.
The new therapy uses exosomes to carry Cas12a into cells to accurately locate the HIV virus, including latent HIV, and shreds its genome, achieving a functional cure for AIDS. It offers advantages such as a strong targeting capability, a high safety level, and the ability to perform multiple collaborative cuts.
In experiments involving HIV-infected mice and blood samples taken from AIDS patients, the therapy demonstrated potent virus clearance and immune reconstitution capabilities. In one experimental group, the complete clearance of the virus was achieved in two out of three mice.
The therapy has passed its medical ethics review and entered the clinical research stage, according to the researchers.