Alternate route on gene therapy raises hope for HIV treatment

Scientists have been toying with zinc finger nucleases for close to two decades now, exploring how ZFN proteins can slice and splice DNA in a surgically precise manner inside the genome. But concerns about the gene therapy methods used to deliver the ZFNs to cells inspired a team of scientists at Scripps Research to find a way to circumvent that approach.

By inserting the ZFN proteins into cells placed in a lab dish, the group found that the ZFNs went right to work, altering the cells without much "collateral damage." Scientists had used a benign virus to transport the ZFNs through cellular membranes, raising the prospect that the viral DNA could implant itself in the cellular DNA. But the unmodified ZFNs used at Scripps worked just fine.

"This work removes a major bottleneck in the efficient use of ZFN proteins as a gene therapy tool in humans," said Michael K. Reddy, who oversees transcription mechanism grants at the National Institutes of Health's National Institute of General Medical Sciences. 

The team now plans to test their new approach on HIV infections, disrupting the CCR5 receptor on T cells to make them resistant to HIV and then re-infusing them back in patients enrolled in a clinical trial.

- here's the press release