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Base editing corrects neurodevelopmental disorder in mice, study shows
Gene editing restores CHD3 function in mice, offering potential treatment for Snijders Blok–Campeau syndrome.
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Gene editing restores CHD3 function in mice, offering potential treatment for Snijders Blok–Campeau syndrome.
A new base-editing therapy corrected a mutation in mice modeling a neurodevelopmental disorder, restoring protein levels and improving symptoms, with promising results in primates.