“The idea of genetically altering people’s cells to make them resist the virus that causes AIDS may seem like a pipe dream, but a new report suggests it can be done.
“The research involves the first use in humans of 'gene editing,' a treatment that zeros in on a particular gene and disables it.”
“On first glance it looks as if this works with the CD4 t-cells while the work we are financing focuses on a similar approach but perhaps the best explanation of the difference comes from this section of the original application from John Zaia's team at City of Hope that is partnering with Sangamo.
“'Recently, ZFNs have been shown to inactivate CCR5 in primary human CD4 T cells, allowing them to preferentially survive and expand in the presence of HIV. A human clinical trial evaluating this approach is on-going, in which patient T cells are re-infused after ZFN-treatment to block CCR5 expression and possibly provide an HIV-resistant reservoir of CD4 T cells (THIS IS THE PIECE IN THE NY TIMES ARTICLE). The CIRM Disease Team proposes an approach to modify a patient’s own HSC to circumvent the need to find matched donors that carry the Δ32 CCR5 mutation and yet provide a renewable and long-lasting source of HIV-resistant cells.'”