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Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

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.

Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.

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

The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.

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.

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.

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.