P2X7 purinoceptor as a target for pharmacotherapy of Duchenne Muscular Dystrophy

Dariusz C Gorecki
University of Portsmouth

27th October 2012

Damaged myofibers, such as those in muscular dystrophy, release ATP into the extracellular space.  Purine nucleotides are recognized as important extracellular signalling molecules and act as agonists for the P2X receptors, including P2X7.   The P2X7 receptor has been referred to as a danger receptor and is up-regulated in mdx muscle.  Reducing P2X7 activity is a proposed target for therapy in Duchenne Muscular Dystrophy.  TACT made recommendations regarding additional preclinical studies to confirm the importance of the P2X7 receptor in Duchenne Muscular Dystrophy and also advised regarding suitable small molecules that may best be able to demonstrate antagonism in the pre-clinical models.  Additionally, TACT provided advice on potential industry relationships to facilitate the preclinical, and ultimately clinical, investigation. TACT also provided guidance on organizing a clinical trial.

12 Apr 2017