ProjectTreatKCNQ - Gezielte Behandlung von KCNQ-Enzephalopathien: Retigabin-Analoga, Wirkstoffrepurposing und…

Basic data

Title:
TreatKCNQ - Gezielte Behandlung von KCNQ-Enzephalopathien: Retigabin-Analoga, Wirkstoffrepurposing und allelspezifischer Knockdown. Teilvorhaben: Validierung von Retigabin- Analoga und Repurposed Compounds in primären Mausneuron- und humanen Hirnschnitt Kulturen
Duration:
6/1/2021 to 5/31/2024
Abstract / short description:
KCNQ-associated encephalopathies (KCNQ-E) are a group of severe epilepsies with onset in the first months of life, characterized by treatment resistant seizures and developmental delay. They are caused by mutations in genes encoding voltage-gated potassium channels that are responsible for the M-current, which plays a critical role in the regulation of neuronal excitability. Both a severe lack and excess of channel function can result in the development of KCNQ-E. Seizures in children with KCNQ-E often respond poorly to anti-epileptic drugs, and more importantly, therapies for the developmental problems are currently unavailable. With the aim to develop improved therapies for KCNQ-E, we will establish a consortium of researchers with expertise in KCNQ-related pathology and drug development, and access to a set of disease-relevant assays and models. We expect to provide pre-clinical evidence for different types of targeted treatments that have the potential to improve the developmental outcome of KCNQ-E.

Involved staff

Managers

Department of Neurosurgery
Department of Neurosurgery and Neurotechnology, Hospitals and clinical institutes, Faculty of Medicine
Department of Neurology and Epileptology
University Department of Neurology, Hospitals and clinical institutes, Faculty of Medicine

Local organizational units

Department of Neurosurgery
Department of Neurosurgery and Neurotechnology
Hospitals and clinical institutes, Faculty of Medicine
Department of Neurology and Epileptology
University Department of Neurology
Hospitals and clinical institutes, Faculty of Medicine

Funders

Bonn, Nordrhein-Westfalen, Germany
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