Topic: lentiviral vectors

Epilepsy is a chronic neurological disorder characterized by recurrent seizures. Despite the availability of many antiepileptic drugs (AEDs), 30% of the patients are resistant to standard therapies. GABAA receptor (GABAAR) expression and function are altered in the epileptic tissue, resulting in impaired inhibition and reduced sensitivity to GABAergic AEDs. Therefore, we are developing a gene therapy strategy for focal drug-resistant epilepsy based on the overexpression of specific GABAAR subunits in hippocampal excitatory neurons, aiming at increasing network inhibition and enhancing the effe

Rett Syndrome (RTT) is a X-linked neurodevelopmental disorder that predominantly affects girls. The detailed disease mechanisms remain unclear and fundamental treatments are yet to be established. The majority of typical RTT cases is triggered by mutations in the methyl CpG-binding protein 2 (MeCP2) gene. For the analyze of the disease mechanisms and drug development, in vitro models based on iPSC-derived cells with diseased MeCP2 are being investigated. In addition to MeCP2 mutations RTT is also affected by the genetic background, and thus it is necessary to analyze the effect of MeCP2 modifi