Elisabeth Manville
Jul 2, 2012

HIV treatment: potential alternative to gene therapy

Scientists have highlighted the potential of a new technique that could act as a safer alternative to an experimental gene therapy against HIV infection. The method involves zinc finger nuclease (ZFN) proteins, which are used as potential disease treatments. ZFN could potentially treat HIV by disrupting the CCR5 gene in T cells. Removing this receptor would make cells highly resistant to HIV infection. The previous DNA-based method that the Scripps Institute team used to deliver the ZFN proteins caused a significant amount of off-target DNA damage. A new direct delivery system, involving simply adding the proteins directly to cells in a lab dish, was shown effective, as the ZFNs crossed into the cells and performed gene-cutting with high-efficiency and minimal unintended damage. This technique could potentially be used in stem cells that could act as miniscule factories to delivery ZFN to cells inside the body.