Dynorphin-Based "Release on Demand" Gene Therapy for Drug-Resistant Temporal Lobe Epilepsy

ABSTRACT: Focal epilepsy represents one of the most common chronic CNS diseases. The high incidence of drug resistance, devastating comorbidities and insufficient responsiveness to surgery pose unmet medical challenges. In the quest of novel, disease-modifying treatment strategies neuropeptides represent promising candidates. Here we provide the "proof of concept" that gene therapy by adeno-associated virus (AAV) vector transduction of preprodynorphin into the epileptogenic focus of well-accepted mouse and rat models for temporal lobe epilepsy leads to suppression of seizures over months. The debilitating long-term decline of spatial learning and memory is prevented. In human hippocampal slices obtained form epilepsy surgery, dynorphins suppressed seizure-like activity, suggestive of a high potenti

SUBMITTER: Prof. Christoph Schwarzer 

PROVIDER: S-SCDT-EMM-2018-09963 | biostudies-other |

REPOSITORIES: biostudies-other