Article
DNA-PKcs inhibitor AZD7648 reveals sgRNA cross-contaminants and enhanced sensitivity of genome engineering off-target activity in HSPCs.
Nucleic acids research - 13 Apr 2026
White Nathan, Hu Yi-Ting, Chalk John Alexander, Kurgan Gavin, Naseem Asma, Schmaljohn Ellen, Sturgeon Morgan, Cavazza Alessia, Thrasher Adrian James, Turchiano Giandomenico
Abstract excerpt
Therapeutic gene editing with designer nucleases can be compromised by undesired repair outcomes. DNA repair inhibitors are used to bias DSB repair toward HDR, but their impact on larger structural rearrangements, including large deletions and translocations, remains unclear. We quantify the mutational burden associated with end-joining inhibitor compounds. With a highly precise Cas9 nuclease, repair inhibition...
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