New developments in the use of gene therapy to treat Duchenne muscular dystrophy.


INTRODUCTION Duchenne muscular dystrophy (DMD) is a lethal X-linked inherited disorder characterised by progressive muscle weakness, wasting and degeneration. Although the gene affected in DMD was identified over 25 years ago, there is still no effective treatment. AREAS COVERED Here we review some of the genetic-based strategies aimed at amelioration of… (More)
DOI: 10.1517/14712598.2014.866087


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