An update on RNA-targeting therapies for neuromuscular disorders.
OBJECTIVE
Antisense-mediated modulation of transcripts is a dynamic therapeutic field, especially for neuromuscular disorders.


RESULTS
For three diseases, this approach has advanced to the clinical trial phase, that is Duchenne muscular dystrophy, spinal muscular atrophy and myotonic dystrophy. In parallel, numerous proof-of-concept studies in cell and animal models have been reported for additional neuromuscular disorders.


CONCLUSIONS
This review discusses the most notable advances in preclinical and clinical studies in the past year. For Duchenne muscular dystrophy, spinal muscular atrophy and myotonic dystrophy trials are ongoing to assess safety and efficacy, while in parallel preclinical studies are being conducted to identify ways to improve efficiency and delivery. For other neuromuscular diseases, progress is made as well warranting future clinical trials. However, towards clinical trial readiness, it is important not only to optimize the therapy preclinically but to also develop the infrastructure that is needed to conduct trials.
Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Silvana
S
Jirka
Annemieke
A
Aartsma-Rus
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009468:Neuromuscular Diseases / Q000628:therapy*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*; D012313:RNA / Q000235:genetics*
D016376:Oligonucleotides, Antisense; D012313:RNA