Colasante, Gaia

dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy 01 2020 - 235-253 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1525-0024

10.1016/j.ymthe.2019.08.018 doi


Action Potentials
Animals
CRISPR-Associated Protein 9--genetics
Cell Line, Tumor
Disease Models, Animal
Epilepsies, Myoclonic--therapy
Female
GABAergic Neurons--metabolism
Genetic Therapy--methods
Hippocampus--cytology
Interneurons--metabolism
Mice
Mice, Inbred C57BL
Mice, Transgenic
NAV1.1 Voltage-Gated Sodium Channel--genetics
Seizures--therapy
Transcriptional Activation
Treatment Outcome