Researchers have used CRISPR to disrupt activity of the mutated gene for lamin A in progeria mice but their health improved only modestly, and disabling a person’s good copy of the gene could cause harm. So David Liu of Harvard University and the Broad Institute turned to base editing, a DNA-changing method originally inspired by CRISPR. Unlike CRISPR, which makes double-strand cuts in DNA, the base editor used in the progeria study nicks just one strand and swaps out a single base. Base editors have treated disorders in individual organs in mice, and Liu wanted to try one on an disease that involves multiple organs or tissues.
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