Scn1a gene reactivation after symptom onset rescues pathological phenotypes in a mouse model of Dravet syndrome

Scn1a gene reactivation after symptom onset rescues pathological phenotypes in a mouse model of Dravet syndrome

Dravet syndrome is a devastating epileptic encephalopathy caused by Scn1a gene haploinsufficiency. Exploiting a novel knock-in mouse model, here the authors show that restoring Scn1a expression after symptom onset is sufficient to rescue main phenotypic manifestations of the syndrome.

Bibliographic Details
Main Authors: Nicholas Valassina, Simone Brusco, Alessia Salamone, Linda Serra, Mirko Luoni, Serena Giannelli, Simone Bido, Luca Massimino, Federica Ungaro, Pietro Giuseppe Mazzara, Patrizia D’Adamo, Gabriele Lignani, Vania Broccoli, Gaia Colasante
Format: Article
Language:English
Published: Nature Portfolio 2022-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-021-27837-w

Internet

https://doi.org/10.1038/s41467-021-27837-w

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