Allele-Specific Editing of a Dominant SCN8A Epilepsy Variant Protects against Seizures and Lethality in a Murine Model.

Wenxi YuSophie F HillYumei HuangLimei ZhuYiannos DemetriouJulie ZiobroFaith RegerXiaoyan JiaJoanna MattisMiriam H Meisler

Published in: Annals of neurology (2024)

The data demonstrate efficient allele-specific editing of a dominant missense variant and support the feasibility of allele-specific therapy for DEE epilepsy. ANN NEUROL 2024.

Keyphrases

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machine learning
intellectual disability
autism spectrum disorder
big data
deep learning
neural network