Article

AAV-mediated interneuron-specific gene replacement for Dravet syndrome

2023-12-15

Abstract excerpt

Dravet syndrome (DS) is a devastating developmental epileptic encephalopathy marked by treatment-resistant seizures, developmental delay, intellectual disability, motor deficits, and a 10-20% rate of premature death. Most DS patients harbor loss-of-function mutations in one copy of SCN1A , which has been associated with inhibitory neuron dysfunction. Here we developed an interneuron-targeting AAV human SCN1A gen...

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Literature Corpus work: 77bf4b32-7a5a-5360-bbe9-25d7f2b87040
DOI: 10.1101/2023.12.15.571820

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