Improper regulation of gene expression underlies a considerable proportion of human disease, yet the majority of therapeutic interventions only target proteins amenable to �small-molecule� or antibody-based inhibition. To extend our therapeutic arsenal to the complete universe of known disease targets requires methodology that can function independently of the nature of the molecular target � a potential promise of tackling disease at the DNA level. Here, we show that control over human genes implicated in disease can be achieved by using an engineered zinc finger protein-based DNA binding domain specific for the gene of interest and a relevant functional domain. Such designed ZFPs exhibit single-gene specificity in the context of the human genome, and function to evoke therapeutic gene control (via the activation, repression, knockout or correction of the targeted gene ) in cellular and animal models of disease.

Control of gene expression with engineered zinc finger proteins

245th National Meeting (2013)

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