Opportunities and challenges for the development of antisense treatment in neuromuscular disorders

Abstract

Introduction: Neuromuscular disorders are diseases of the musculature and/or the nervous system, generally leading to loss of muscle function. They are a frequent cause of disability and treatment options are often only symptomatic. Interestingly, for a number of neuromuscular disorders the application of antisense oligonucleotides has therapeutic potential.

Areas covered: The authors describe how this approach is exploited for different neuromuscular diseases, focusing on literature published in the past 10 years. For each disease the opportunities of this approach, the state of the art, and current challenges are described.

Expert opinion: A lot of progress has been made in the development of antisense-mediated approaches during recent years and they may become clinically applicable in the near future.

Keywords::

• antisense oligonucleotides
• exon skipping
• neuromuscular disorders
• therapy

Notes

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