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dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice

Molecular Therapy : the Journal of the American Society of Gene Therapy

Oct 15, 2019

Gaia ColasanteVania Broccoli

Abstract

Dravet syndrome (DS) is a severe epileptic encephalopathy caused mainly by heterozygous loss-of-function mutations of the SCN1A gene, indicating haploinsufficiency as the pathogenic mechanism. Here we tested whether catalytically dead Cas9 (dCas9)-mediated Scn1a gene activation can resc...read more

Mentioned in this Paper

Voltage-Gated Sodium Channels
Genes
Infantile Severe Myoclonic Epilepsy
Adeno-Associated Virus
Transcription, Genetic
Neurons
Gene Mutation
SCN1A gene
Encephalopathic Epilepsy
Interneurons
34
19
1
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dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice

Molecular Therapy : the Journal of the American Society of Gene Therapy

Oct 15, 2019

Gaia ColasanteVania Broccoli

PMID: 31607539

DOI: 10.1016/j.ymthe.2019.08.018

Abstract

Dravet syndrome (DS) is a severe epileptic encephalopathy caused mainly by heterozygous loss-of-function mutations of the SCN1A gene, indicating haploinsufficiency as the pathogenic mechanism. Here we tested whether catalytically dead Cas9 (dCas9)-mediated Scn1a gene activation can resc...read more

Mentioned in this Paper

Voltage-Gated Sodium Channels
Genes
Infantile Severe Myoclonic Epilepsy
Adeno-Associated Virus
Transcription, Genetic
Neurons
Gene Mutation
SCN1A gene
Encephalopathic Epilepsy
Interneurons
34
19
1

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Paper Details
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