Using engineered zinc-finger nucleases—DNA-binding proteins that target specific sequences—researchers at a biotechnology company report this week in an advanced online publication in Nature that they have achieved the highest levels of permanent targeted gene correction in cultured human cells described to date.

Michael Holmes and his colleagues at Richmond, Ca.–based Sangamo Biosciences corrected a defective version of the IL2R-gamma receptor gene, which is mutant in children with X-linked severe combined immunodeficiency. Sangamo had used zinc-finger proteins before in several instances, among them, to inhibit specific single gene expression in vivo.

"With our technology platform here at Sangamo, we can basically engineer zinc-finger proteins to recognize virtually any sequence," Holmes, the senior author of the study, told The Scientist. "Here, we show that gene correction can be done at a specific site of the genome, where a mutation occurs, and at high frequency."

The authors report that...

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