Gene editing using adenine base editing repaired a DNA error causing Dravet syndrome in mice, resulting in far fewer seizures and significantly longer survival.
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Medicine Breakthroughs Medicine Breakthroughs Health
Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study

Far Fewer After Editing Seizure frequency
Significantly Longer Survival
Single Brain Injection Delivery method
Adenine Base Editor Gene editing tool
SCN1A R613X Target variant
Day 1 Or Day 12 injection timing
15,000–20,000 US patients affected