Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy.

ABSTRACT

Duchenne muscular dystrophy (DMD) is a serious genetic neuromuscular rare disease that is prevalent and caused by the mutation/deletion of the X-linked DMD gene that encodes dystrophin. Utrophin is a dystrophin homologous protein on human chromosome 6. Dystrophin and utrophin are highly homologous. They can recruit many dystrophin-glycoprotein complex (DGC)-related proteins and co-localize at the sarcolemma in the early stage of human embryonic development. Moreover, utrophin is overexpressed naturally at the mature myofiber sarcolemma in DMD patients. Therefore, utrophin is considered the most promising homologous protein to replace dystrophin. This review summarizes various modulating drugs and gene therapy approaches for utrophin replacement. As a universal method to treat DMD disease, utrophin has a promising therapeutic prospect and deserves further investigation.