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Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntington’s disease and Friedreich’s ataxia.

The new system is the first to use a DNA-mobilizing enzyme called a CRISPR-associated transposase to make targeted gene-sized edits at therapeutically useful levels in human cells.

Liu is honored for the development of base editing and prime editing, two gene editing technologies transforming medicine.

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.