A Review of Targeted Therapies for Monogenic Epilepsy Syndromes

Vincent Zimmern, Berge Minassian, Christian Korff

Research output: Contribution to journalReview articlepeer-review

Abstract

Genetic sequencing technologies have led to an increase in the identification and characterization of monogenic epilepsy syndromes. This increase has, in turn, generated strong interest in developing “precision therapies” based on the unique molecular genetics of a given monogenic epilepsy syndrome. These therapies include diets, vitamins, cell-signaling regulators, ion channel modulators, repurposed medications, molecular chaperones, and gene therapies. In this review, we evaluate these therapies from the perspective of their clinical validity and discuss the future of these therapies for individual syndromes.

Original languageEnglish (US)
Article number829116
JournalFrontiers in Neurology
Volume13
DOIs
StatePublished - Feb 17 2022

Keywords

  • anti-sense oligonucleotide
  • channelopathy
  • gene therapy
  • genetic epilepsy
  • ketogenic diet
  • molecular chaperone

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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