ABSTRACT
Utrophin in the Therapy of Duchenne Muscular Dystrophy Q ing Bai, Edward A. Burton and K ay E. D avies
Duchenne muscular dystrophy (DMD) is a devastating muscle wasting disease caused by the lack of the cytoskeletal protein dystrophin in muscle. The dystrophin-related protein, utrophin, shows a high degree of sequence similarity to dystrophin and it has been postulated that utrophin replacement could be a possible therapeutic strategy for the disease. We review the evidence that utrophin can functionally replace utrophin and the work being carried out on the transcriptional regulation of the gene.
