This month, Dr. David Liu at the Broad Institute of MIT and Harvard in Cambridge, MA, introduced a CRISPR upgrade that in theory may correct nearly 90 percent of all disease-causing genetic variations
Rather than simply deactivating a gene, CRISPR-based “prime editing” is a true search-and-replace editor for the human genome. With a single version, it can change individual DNA letters, delete letters, or insert blocks of new letters into the genome, with minimal damage to the DNA strand.
For now, prime editing has only been tested in cultured cells. But its efficacy is off the charts. Early experiments found it could correct single-letter misspellings in sickle cell disease, snip out four superfluous letters that underlie Tay-Sachs, and insert three missing letters to correct a genomic typo that leads to cystic fibrosis. In all, the tool worked remarkably well in over 175 edits in both human and mouse cells.
“The excitement has been palpable,” said Dr. Fyodor Urnov at the University of California, Berkeley, who was not involved in the research. “I can’t overstate the significance of this.”
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