Koo T., Linda Popplewell None., Alberto Malerba None., George Dickson None.
This chapter focuses on the gene therapy advances made in relation to Duchenne muscular dystrophy and discusses principles and perspectives of strategies currently being developed. The chapter explains the genetic mutations that cause Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) and the differences between the two are discussed in relation to disease severity. The histopathological features of DMD are explained and discussed in the context of available animal models for DMD. There are various genetic therapeutic options available for the treatment of DMD, and the progress of each therapeutic approach is promising. A number of specific areas for the treatment of DMD are comprehensively presented, alongside in-depth description of the genetic biology of muscular dystrophy.