Article
CRISPR/dCas9-based Scn1a gene activation in inhibitory neurons ameliorates epileptic and behavioral phenotypes of Dravet syndrome model mice.
Neurobiology of disease - 1 Jul 2020
Yamagata Tetsushi, Raveau Matthieu, Kobayashi Kenta, Miyamoto Hiroyuki, Tatsukawa Tetsuya, Ogiwara Ikuo, Itohara Shigeyoshi, Hensch Takao K, Yamakawa Kazuhiro
Abstract excerpt
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 RNAs (gRNAs) in the promoter regions together with the nuclease-deficient Cas9 fused to transcription activators...
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