The role of dystrophin in the central nervous system: a mini review.

: Dystrophin is a protein responsible for a severe muscle disease Duchenne muscular dystrophy (DMD). This molecule, a huge 427 kDa protein, is also expressed in brain neurons, but its function in the central nervous system (CNS) has been obscure. It is known that some DMD patients have accompanying symptoms of CNS disorders such as the cognitive impairment or psychiatric disorders. The bases of the CNS symptoms are unknown. Addressing the CNS function of dystrophin would make the advance on the interpretation of the CNS symptoms accompanying with DMD. In this short review, previous findings on dystrophin in the CNS are summarized to facilitate studies on the CNS function of this molecule. Following topics are in particular focused, (1) regional and subcellular distribution of dystrophin in the brain and the function in GABAergic synapses, (2) the CNS abnormality in dystrophin-deficient 'mdx' mice.