Viltolarsen for the treatment of Duchenne muscular dystrophy.
Antisense therapy
Duchenne muscular dystrophy
Gene therapy
NS-065/NCNP-01
Oligonucleotides
Phosphorodiamidate morpholino oligomers
Viltolarsen
Journal
Drugs of today (Barcelona, Spain : 1998)
ISSN: 1699-3993
Titre abrégé: Drugs Today (Barc)
Pays: Spain
ID NLM: 101160518
Informations de publication
Date de publication:
Oct 2019
Oct 2019
Historique:
entrez:
14
11
2019
pubmed:
14
11
2019
medline:
26
11
2019
Statut:
ppublish
Résumé
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 oligonucleotides 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.
Identifiants
pubmed: 31720560
pii: 3045038
doi: 10.1358/dot.2019.55.10.3045038
doi:
Substances chimiques
Dystrophin
0
Morpholinos
0
Oligonucleotides
0
Oligonucleotides, Antisense
0
RNA Precursors
0
viltolarsen
SXA7YP6EKX
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
627-639Informations de copyright
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