Therapeutic modalities targeting RNA transcripts or delivering RNA to cells have therapeutic potential for neuromuscular diseases. For RNA targeting, antisense oligonucleotides have received marketing authorization for a number of genetic and acquired neuromuscular diseases and many clinical trials are ongoing to evaluate more. However, for both oligonucleotides and mRNA therapies there is clear room for improvement with regards to delivery.
To commemorate the successes, but also to highlight the cutting-edge science that is ongoing in this field, the Journal of Neuromuscular Diseases will have a special issue on “RNA therapeutics in neuromuscular disorders”. For this issue we welcome both review papers and original research contributions. If you are considering to submit a review, please align first with the guest editors about the proposed topic.
The publication charge is waived for this special issue.
Annemieke Aartsma-Rus, PhD, Professor of Translational Genetics, Dept of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, the Netherlands. Email: a.m.rus@lumc.nl
Shin'ichi Takeda, MD, PhD, National Institute of Neuroscience, Translational Medical Center Tokyo, Japan. Email: takeda@ncnp.go.jp
