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DOI: 10.1055/a-1995-5405
Gentherapien für Epilepsie: Klinische Studien sind auf dem Weg
Gene Therapy for Epilepsy: Clinical Studies are on the Road Funding Dieser Beitrag wurde unterstützt von der Deutschen Forschungsgemeinschaft (DFG, FOR-2715/2, Le1030/15-2, Le1030/23-1), dem Bundesministerium für Bildung und Forschung (BMBF, Treat-ION, 01GM2210A) und der Else-Kröner-Fresenius-Stiftung (Forschungskolleg Precise.net).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 EpilepsieKey words
epilepsy - gene therapy - neurodevelopmental disorder - developmental and epileptic encephalopathy - Refractory focal epilepsyPublication History
Received: 05 October 2022
Accepted: 27 November 2022
Article published online:
30 January 2023
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