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The base editing enzyme used in this study is the same one that fixed Baby KJ Muldoon’s disease-causing mutation and saved his life in 2025.

Researchers have developed a genome-editing strategy that targets a common cause of roughly 30 percent of rare diseases and could vastly improve access to gene-editing treatments for patients.

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.

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 engineered a compact degron tag that can be precisely edited into a cell’s genome, enabling scientists to trigger the rapid breakdown of native target proteins.

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.