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The approach targets the most common genetic cause of the disease and could enable a one-time treatment as effective as existing daily therapies.

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.

Researchers have evolved the editing machine at the heart of the prime editing system, which can make a wide range of changes to the genome.

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.

Greg Newby discusses the value of mentorship and his goals for his next career step.