CRISPR/dCas9-based Scn1a gene activation in inhibitory neurons ameliorates epileptic and behavioral phenotypes of Dravet syndrome model mice

CRISPR/dCas9-based Scn1a gene activation in inhibitory neurons ameliorates epileptic and behavioral phenotypes of Dravet syndrome model mice

Dravet syndrome is a severe infantile-onset epileptic encephalopathy which begins with febrile seizures and is caused by heterozygous loss-of-function mutations of the voltage-gated sodium channel gene SCN1A. We designed a CRISPR-based gene therapy for Scn1a-haplodeficient mice using multiple guide...

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Bibliographic Details
Main Authors: Tetsushi Yamagata, Matthieu Raveau, Kenta Kobayashi, Hiroyuki Miyamoto, Tetsuya Tatsukawa, Ikuo Ogiwara, Shigeyoshi Itohara, Takao K. Hensch, Kazuhiro Yamakawa
Format: Article
Language:English
Published: Elsevier 2020-07-01
Series:Neurobiology of Disease
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996120302291

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http://www.sciencedirect.com/science/article/pii/S0969996120302291

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