Article

AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome

2024-09-28

Abstract excerpt

Dravet syndrome (DS) is a severe childhood onset developmental and epileptic encephalopathy which leads to life-long disability. Symptoms usually manifest in the first year of life and include prolonged severe seizures, developmental delay and severe intellectual disability. DS patients have an increased mortality rate, including sudden unexpected death in epilepsy (SUDEP). Approximately 90% of patients carry a he...

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Literature Corpus work: 7ec49921-508c-57df-8dc7-bf671bdebf7e
DOI: 10.1101/2024.09.27.615323

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