Herein, we have studied the potential of nanomedicineto prepare a single platform based on mesoporous silica nanoparticles(MSNs) for the treatment of an ALS animal model with a cocktail ofagents such as leptin (neuroprotective) and pioglitazone (anti-inflammatory),which have already demonstrated promising therapeutic ability in otherneurodegenerative diseases. The gene discussed is LEP; the disease is amyotrophic lateral sclerosis.