New CRISPR tool has the potential to correct almost all disease-causing DNA glitches, scientists report
New CRISPR tool
has the potential to correct almost all disease-causing DNA glitches,
A new form of the genome-editing tool CRISPR-Cas9 appears to significantly expand the range of diseases that could be treated with the technology, by enabling scientists to precisely change any of DNA’s four “letters” into any other and insert or delete any stretch of DNA — all more efficiently and precisely than previous versions of CRISPR. Crucially, scientists reported on Monday, it accomplishes all that without making genome-scrambling cuts in the double helix, as classic CRISPR and many of its offshoots do.
News about this “prime editing” began circulating among CRISPR-ites this month, when the inventors unveiled it at a meeting at Cold Spring Harbor Laboratory. Since then, “the excitement has been palpable,” said genetic engineer Fyodor Urnov of the University of California, Berkeley, who was not involved in the research.
“I can’t overstate the significance of this,” he said, likening the creation of ever-more kinds of genome-editing technologies to the creation of superheroes with different powers: “This could be quite a useful Avenger for the genome-editing community, especially in translating basic research to the clinic” to cure diseases ranging from sickle cell to cystic fibrosis.
Prime editing’s inventors, led by David Liu of the Broad Institute of MIT and Harvard and postdoctoral fellow Dr. Andrew Anzalone, say it has the potential to correct 89% of known disease-causing genetic variations in DNA, from the single-letter misspelling that causes sickle cell to the superfluous four letters that cause Tay-Sachs disease. All told, they report making 175 edits in human and mouse cells.