Journal
EUROPEAN JOURNAL OF HUMAN GENETICS
Volume 18, Issue 2, Pages 146-153Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/ejhg.2009.160
Keywords
Duchenne muscular dystrophy; spinal muscular atrophy; myotonic dystrophy; therapy; antisense oligonucleotide; exon skipping
Funding
- ZonMw (the Netherlands)
- Duchenne Parent Project (the Netherlands)
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Neuromuscular disorders are a frequent cause of chronic disability in man. They often result from mutations in single genes and are thus, in principle, well suited for gene therapy. However, the tissues involved (muscle and the central nervous system) are post-mitotic, which poses a challenge for most viral vectors. In some cases, alternative approaches may use small molecules, for example, antisense oligonucleotides (AONs). These do not deliver a new gene, but rather modulate existing gene products or alter the utilization of pathways. For Duchenne muscular dystrophy, this approach is in early phase clinical trials, and for two other common neuromuscular disorders (spinal muscular atrophy and myotonic dystrophy), significant preclinical advances have recently been made. European Journal of Human Genetics (2010) 18, 146-153; doi:10.1038/ejhg.2009.160; published online 7 October 2009
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