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Background CCR5 is the major HIV co-receptor, and individuals homozygous for a 32-bp deletion in CCR5 are resistant to infection by CCR5-tropic HIV-1. Zinc finger nuclease (ZFN) technology is a class of engineered DNA-binding proteins facilitating targeted genome editing by binding to a user-specific locus and causing a double-strand break in the region of interest. As a result, the gene of interest targeted by ZFN cleavage is disrupted. We investigated the ability of a Ccr5 gene-specific ZFN to establish SIV-resistant CD4+ T cells and hematopoietic progenitor cells isolated from macaques.

Engineering SIV-resistant macaque hematopoietic stem cells and CD4+ T cells with CCR5-specific zinc-finger nucleases