Therapeutic strategies for Hereditary Spastic Paraplegia

HSP are still considered incurable. Treatment options are essentially limited to physiotherapy. To date, no causal (gene) therapy for HSP exists. In recent years, various molecular tools and methods for the effective application of gene therapy have been developed and established. Regarding HSP, there is only one study showing that the viral-based transfer of intact cDNA blocks progressive axon degeneration of lower motor neurons (peripheral nervous system, PNS) in a murine disease model. Somatic gene repair studies targeting the CNS as the target site of primary HSP pathology are currently not available. It remains unclear whether the therapeutic approach of somatic gene repair in hereditary degenerative motor neuron diseases has any effect on the potentially pre-damaged whole organism or on potentially pre-damaged individual organ systems such as the CNS and its motor neurons. To address this question, it is inevitable to analyze novel disease models that we already developed in our laboratory.