Article
New developments in the use of gene therapy to treat Duchenne muscular dystrophy.
Expert opinion on biological therapy - 1 Feb 2014
Jarmin Susan, Kymalainen Hanna, Popplewell Linda, Dickson George
Abstract excerpt
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 the DMD phenotype. A number of Phase II/III clinical...
Topics
- Animals
- Clinical Trials, Phase II as Topic
- Clinical Trials, Phase III as Topic
- Codon, Terminator
- Dependovirus
- Dystrophin
- Exons
- Gene Transfer Techniques
- Genetic Therapy
- Genetic Vectors
- Humans