FAU own research funding: EFI / IZKF / EAM ...
Start date : 01.04.2023
End date : 15.06.2023
Pathogenic variants in SPAST, which encodes spastin, a microtubule-severing enzyme, are the most common cause of Hereditary spastic paraplegia, a motor neuron disorder affecting the axons of corticospinal motor neurons. To date, therapeutic strategies focus on ameliorating the symptoms without treating the underlying cause. Here we investigate the effects of microtubule modifying drugs on SPAST induced pluripotent stem cell derived neurons.