Investigators at Harvard say that they have successfully used CRISPR Cas gene-editing tools to come up with a new approach in curing HIV, a lethal virus that has claimed millions of lives over recent decades. And they're prepping for animal studies now to see if they can provide some preclinical proof-of-concept data to back up their lab experiment.
CRISPR Cas--clustered regularly interspaced short palindromic repeat-activated cells--has been gaining a foothold in the biotech world over the past couple of years. Chad Cowan and Derrick Rossi at the Harvard Stem Cell Institute are working to eliminate the CCR5 receptor in T cells, the back door that HIV uses to strip the body of immune protection.
The big idea here is that CRISPR Cas could be used to create resistant blood stem cells in bone marrow and then transplant the bone marrow into humans suffering from HIV. Then the HIV-proof immune system T cells can durably fight back against the HIV.
"We showed that you can knock out CCR5 very efficaciously, we showed that the cells are still functional, and we did very, very deep sequencing analysis to show that there were no unwanted mutations, so it appears to be safe," Cowan told the Harvard Gazette. He added that "there is obviously much more work to do."
If the investigators can gain the animal data they're looking for, then they'll look for regulatory approval to try it in humans. The theory has already been tested in one "Berlin patient" who was able to fight off HIV after having bone marrow from an HIV-resistant individual with the rare genetic defect that eliminated CCR5 receptors from his T cells.
CRISPR Therapeutics in Basel and Editas Medicine in Cambridge, MA, have both been launched fairly recently to develop new therapies using CRISPR Cas.
- here's the story from the Harvard Gazette