Emerging Genetic Therapies in Epilepsy

Abstract

Advances in next-generation sequencing have allowed for genetic characterization of many epilepsies that previously had no known cause. Although having a precise diagnosis has been very helpful for management, counseling, and prognosis, until recently true precision medicine approaches that could have disease-modifying effects have been lacking. This review will highlight how preclinical animal models of Dravet syndrome have allowed some novel gene therapy approaches to be studied and further developed such that they are now entering into the clinics.

Keywords

epilepsy - gene therapy - Dravet syndrome - antisense oligonucleotide - ASO - AAV9

Publikationsverlauf

Accepted Manuscript online:
24. Februar 2025

Artikel online veröffentlicht:
24. März 2025

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