The gene-editing approach uses prime editors and evolved enzymes called recombinases, and could pave the way to effective one-size-fits-all gene therapies for diseases such as cystic fibrosis.
By adapting virus-like particles to carry the machinery for a type of gene editing called prime editing, scientists have corrected disease-causing mutations in animals and increased editing efficiency.
Scientists at St. Jude Children’s Research Hospital and the Ó³»´«Ã½ 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.