Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard showed how prime editing can correct mutations that cause sickle cell disease in a potentially curative approach.
A one-time genome-editing treatment restores motor function and extends lifespan in an animal model of a neuromuscular disease that is the leading genetic cause of infant mortality.
New method finds compounds that bind and inhibit individual members of a family of key regulatory proteins, called cyclophilins, that have been difficult to target selectively.
Researchers have boosted the efficiency of prime editing, a highly versatile CRISPR-based gene editing technology, and used the improved system to correct disease mutations in cells.