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Intravenous injections AAV particles containing gRNAs mice Scn1a-haplodeficiency neuron-specific dCas9-VPR inhibits SCN1A. 1 / 1

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"Intravenous injections of AAV particles containing the optimal combination of 4 gRNAs into transgenic mice with Scn1a-haplodeficiency and inhibitory neuron-specific expression of dCas9-VPR at four weeks of age increased Nav1.1 expression in parvalbumin-positive GABAergic neurons , ameliorated their febrile seizures and improved their behavioral impairments ."

32445790

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IndraLab

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Intravenous injections AAV particles containing gRNAs mice Scn1a-haplodeficiency neuron-specific dCas9-VPR inhibits SCN1A. 1 / 1

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