NOVEL MICRO-RNA THERAPY FOR TREATMENT OF DRAVET SYNDROME, A PEDIATRIC ORPHAN DISEASE

Dravet syndrome (DS), also known as severe myoclonic epilepsy of infancy (SMEI), is a genetic and rare form of treatment-refractory epilepsy that begins in infancy. DS incidence is estimated at 1/30,000 with about 20,000-40,000 patients worldwide. The long-term prognosis for DS patients is poor, including cognitive impairment, behavioral problems, and a high incidence of sudden death in childhood.

Dr. Schaefer and colleagues have discovered that the micro-RNA, miR-128, is a master regulator of neuronal excitability and motor function. Neuron-specific miR-128 deficiency in mice invariably resulted in seizure-induced death (Fig A) and