Pharmacological actions and potential therapeutic use of cannabinoids in Duchenne's muscular dystrophy

The scientific community uses the term endocannabinoid system (ECS) to refer to a large group of molecules that in our body control the production and function of the two major cannabinoid lipid-mediators, named Anandamide (AEA) and 2-Arachidonoylglycerol (2-AG). Following their discovery, an impressive number of studies have shown that both AEA and 2-AG play a key role in a large plethora of functions in living organisms. Consequently, functional impairment or dysregulation of AEA and 2-AG activity leads to a variety of disorders affecting the nervous system as well as peripheral organs and tissues. For this reason, cannabinoids and/or cannabinoid synthetic drugs currently represent an important area of research for their potential therapeutic use to treat many human diseases having or not a genetic component. Despite these evidences, the role of the endocannabinoid system and hence potential changes in its activity in inherited muscular dystrophies remains largely unknown. Only recently, the role of endocannabinoid CB1 receptors was identified in Duchenne's muscular dystrophy (DMD). In this chapter, I summarize the chemical properties and functional role of the endocannabinoids as well as plant-derived cannabinoids during skeletal muscle formation and repair under physiological conditions as well as DMD.