Inflammation as a possible target in the treatment of SPG11: repurposing of clinically established immunomodulators for the treatment of a genetic disease of the nervous system

Subproject 9

Project lead: Rudolf Martini (Universität Würzburg)


In mouse models for SPG11, we want to investigate whether genetic inactivation of the adaptive immune system leads to a mitigation of the disease. If confirmed, the next step would be to investigate the effect of immunomodulators originally developed for the much more common disease multiple sclerosis. Preventive and therapeutic treatment regimes will be applied. Furthermore, the consequences of treatment discontinuation will be investigated. In addition to histopathological studies, non-invasive, longitudinal measurement methods (e.g. optical coherence tomography, movement analyses, behavioral tests) will be applied to simulate a situation as close to clinical practice as possible. Ideally, our study will provide the basis for a realistic SPG11 treatment concept.