HIV News: Gene therapy stops HIV in mice

There is no HIV cure today.  But new research released online July 2 in Nature Biotechnology suggests that a gene therapy based on genetically modified stem cells may someday serve as one effective HIV treatment to stop HIV, which causes AIDS, from attacking cells.

For the research, Dr. Paula Cannon at the University of Southern California and colleagues genetically manipulated human stem cells and then inserted them into mice where they multiplied into a great number of immune system cells protecting effectively against infection with HIV.

This was observed in humanized mice that had human immune systems and resisted human disease. It remains known however how this so-called gene therapy would work in humans. Cannon was cited by healthday.com as saying that four years of work may be needed before human trials can begin.

What the researchers did is essentially disable a gene called CCR5 that is associated with a receptor of immune cells that HIV needs for infection. Without this receptor, HIV simply can't land on cells and enter them.

In the study, Cannon and colleagues inserted genetically modified stem cells into the humanized mice and other mice as control and then infected all of them with HIV.

As a result, the genetically engineered stem cells produced a great number of mature immune system cells like T-cells in the humanized mice a couple of weeks later that appeared to protect against HIV infection.

In comparison, HIV had no problem attacking other mice that were not treated as the humanized mice.

Cannon was cited as saying if this gene therapy proves effective in humans, the one-time HIV treatment may cost $100,000.  Although costly, it may be better than taking antiviral drugs for the rest of patients' lives.

More than one million people live with HIV and half a million people have died from AIDS in the United States, according to avert.org. 

By Jimmy Downs