Biologists tend not to discuss experimental results on a handful of cells and a single solitary mouse — too preliminary, too sketchy. David Liu of the Broad Institute therefore had no plans to present such findings, which he’d peeked at over his graduate student’s shoulder, when he gave a high-profile talk in 2018 at the National Institutes of Health on a form of the CRISPR genome-editing system that he’d invented.
Not that he wasn’t tempted. Student Luke Koblan had used the clever new form of CRISPR, called base editing, to alter a single misspelled pair of “letters” among the 3 billion in the DNA of cells taken from children with progeria, an infamous and fatal genetic disease marked by accelerated aging. Koblan had done this work in lab dishes, and had also corrected the progeria mutation in a mouse carrying the human gene that, as a result, aged so quickly that by toddlerhood, it was like a picture of Dorian Gray with whiskers.
Chatting before his talk with NIH Director Francis Collins, who discovered the progeria mutation in 2003, Liu happened to mention the results. Collins was blown away. You have to put that in your talk, Collins said. When the head of the NIH speaks, biologists listen — and in Liu’s case, run to the men’s room to update his talk with how a CRISPR base editor might just be the long-sought cure to progeria. Not a treatment, like the drug lonafarnib that was approved this past November, but a one-and-done cure.
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