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• W2184061570 abstract "Clinical applications need targeting of single nucleotide modifications for the treatment of genetic diseases caused by point mutations. Advances in randomized production and targetoriented selection of engineered DNA binding domains have incited a rapidly expanding field of designer transcription factors (TFs). Engineered (TFs) are used in zinc finger nuclease (ZFN) technology that allows targeted genome editing. ZFNs consist of an engineered zinc-finger binding domain that stimulates gene repair by homologous recombination. Zinc finger binding domains fabricated by modular assembly display an unexpectedly high failure rate, having either a lack of activity as ZFNs in human cells, or activity at “off-target” binding sites on the human genome causing cell death. The biological functions of DNA-binding proteins require that they interact with their target binding site with high specificity and affinity. To address these shortcomings we present a theoretical framework to predict highly active ZFN domains. The theoretical framework may enable revisiting the specificity of experimental recognition of zinc fingers toward trinucleotides, and thus provide clinical applications in gene regulations and genome modifications. We produced the entire binding profile of 64 combinations of trinucleotides of the 2nd finger of SP1, and used the data set to predict highly active ZF domains." @default.
• W2184061570 created "2016-06-24" @default.
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• W2184061570 date "2015-01-01" @default.
• W2184061570 modified "2023-09-27" @default.
• W2184061570 title "Theoretical Framework for predicting highly active Zinc Finger Domains" @default.
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