Sangamo readies HIV tech for human trials

Sangamo BioSciences has reported new work in Nature Biotechnology demonstrating the effectiveness of its zinc finger DNA-binding proteins in immunizing cells in the immune system against HIV. A one-time exposure to Sangamo's ZFN approach can change the DNA sequence of the CCR5 gene to stop the spread of the virus. While there are currently available drugs that block HIV from binding to the CCR5 gene, they have to be regularly administered and patients frequently suffer side effects.

"A ZFN approach represents the 'next generation' of HIV-entry blocking agents and a potentially promising class of anti-HIV compounds," said Dr. Carl June, director of translational research at the Abramson Family Cancer Research Institute and the senior author of the study. "These proof of principle data, together with experience from individuals that carry a natural mutation in their CCR5 gene suggest that permanent 'knock-out' of the of CCR5 gene is important and clinically relevant for long-term resistance to HIV infection and, we believe, may prove to be more effective than temporary 'knock down' approaches based on small molecule inhibitors, antibodies, antisense or RNAi."

A human trial of the process is now being readied.

- see this release
- check out the AP report