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Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study
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Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study
Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer. Published in Science Translational Medicine, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic …
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