Gene editing repairs Dravet syndrome in mice, offering hope for rare childhood epilepsy

Dravet syndrome affects an estimated 15,000-20,000 patients in the United States, causing drug-resistant seizures, developmental impairments, and high risk of sudden unexpected death in children.

In the long human struggle against diseases written into our very DNA, a team of researchers at The Jackson Laboratory has demonstrated something quietly extraordinary: a single injection of gene-editing machinery into a newborn mouse's brain can correct the genetic misspelling that causes Dravet syndrome, a rare and often fatal childhood epilepsy. Using a technique called adenine base editing—which rewrites a single DNA letter without severing the genome's architecture—scientists corrected nearly 60 percent of the mutated DNA, dramatically reducing seizures and extending survival. For the est

Gene editing repairs Dravet syndrome in mice, offering hope for rare childhood epilepsy

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