Sangamo Therapeutics, Inc., a genomic medicine company, announced today the publication in Nature Biotechnology of a manuscript by Jeffrey Miller, Ph.D., and colleagues at Sangamo, describing two new strategies for optimizing the specificity of genome editing using zinc finger nucleases (ZFNs). The ability to engineer highly specific gene editing nucleases with little or no detectable activity at unintended genomic sequences is a key safety factor for therapeutic applications. The strategies entail engineering the two key functional regions within the ZFN structure, namely adjusting the binding affinity of the zinc finger array which recognizes DNA, and slowing the catalytic rate of the Fok1 cleavage domain. The two approaches, which are complementary, may be combined to enable near 100% on-target modification with no detectable off-targets. The manuscript, titled “Enhancing gene editing specificity by attenuating DNA cleavage kinetics,” was published online on July 29 and will appear in the August issue of Nature Biotechnology.
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Source: Phys.org