Development of neuron-derived, mitochondria-containing extracellular vesicles for ALS treatment

Introduction: Amyotrophic Lateral Sclerosis (ALS) is a neurological disorder where mitochondrial dysfunction accelerates motor neuron degeneration [1]. Current FDA-approved therapeutics for ALS target individual events of mitochondrial dysfunction [2, 3] but this may not be sufficient to restore the function of damaged motor neurons. A novel approach to mitigate mitochondrial dysfunction is to deliver functional mitochondria to the damaged motor neurons. Therefore, we investigated the potential of neuron-derived mitochondria containing extracellular vehicles (EVs) as a novel therapeutic approach for ALS.