期刊
DRUGS OF TODAY
卷 55, 期 10, 页码 627-639出版社
PROUS SCIENCE, SAU-THOMSON REUTERS
DOI: 10.1358/dot.2019.55.10.3045038
关键词
Viltolarsen; NS-065/NCNP-01; Duchenne muscular dystrophy; Antisense therapy; Oligonucleotides; Phosphorodiamidate morpholino oligomers; Gene therapy
资金
- Muscular Dystrophy Canada
- Friends of Garrett Cumming Research Fund
- HM Toupin Neurological Science Research Fund
- Canadian Institutes of Health Research (CIHR)
- Alberta Innovates: Health Solutions (AIHS)
- Jesse's Journey
- Women and Children's Health Research Institute (WCHRI)
Duchenne muscular dystrophy is the most common lethal X-linked genetic disorder, characterized by progressive muscle loss, with cardiac and respiratory complications. It is caused by a lack of dystrophin protein due to mutations in the DMD gene, which can disrupt the reading frame of the dystrophin primary transcript. Antisense oligonu-cleotides such as phosphorodiamidate morpholino oligomers (PMOs) can induce exon skipping during pre-mRNA splicing and restore the reading frame of the DMD primary transcript. The resulting dystrophin protein is internally deleted but partially functional. Viltolarsen, also known as NS-065/NCNP-01, is a PMO developed through comprehensive sequence optimization and is designed to skip exon 53 on the DMD primary transcript. Exclusion of exon 53 from the DMD primary transcript can treat 8-10% of DMD patients worldwide. This review paper summarizes the mechanism of action, pharmacokinetics and safety of viltolarsen from preclinical and clinical trials.
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