Gentherapien für Epilepsie: Klinische Studien sind auf dem Weg

Zusammenfassung

Seit über 10 Jahren wird an Gentherapien für die schwersten Formen von Epilepsie geforscht, die bis jetzt therapieresistent sind. Nun ergeben sich für fokale pharmakoresistente Epilepsien und für das Dravet Syndrom Gentherapieansätze in ersten klinischen Studien. In diesem Artikel beschreiben wir die Funktionsweise und Ziele dieser und weiterer Gentherapien.

Abstract

For more than 10 years, research has been conducted on gene therapies for the most severe forms of epilepsy, which until now have proven resistant to treatment. First gene therapies are now in clinical trials for pharmacoresistant focal epilepsies and Dravet syndrome. In this article, we describe how these and many more gene therapies work and what they target.

Schlüsselwörter

Epilepsie - Gentherapie - entwicklungsneurologische Störung - entwicklungssassoziierte und epileptische Enzephalopathien - Therapie-refraktäre Epilepsie

Key words

epilepsy - gene therapy - neurodevelopmental disorder - developmental and epileptic encephalopathy - Refractory focal epilepsy

Publication History

Received: 05 October 2022

Accepted: 27 November 2022

Article published online:
30 January 2023

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