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Synonyms: SRP-4053 | SRP4053 | VYONDYS 53®
golodirsen is an approved drug (FDA (2019))
Compound class:
Nucleic acid
Comment: Golodirsen (SRP-4053) is an antisense RNA oligonucleotide that uses the phosphorodiamidate morpholino oligomer (PMO) chemistry [2]. It was designed to induce exon 53 skipping in the dystrophin gene as a mechanism to increase expression of functional dystrophin protein in the muscle fibers of appropriately selected Duchenne muscular dystrophy patients [1].
Full sequence as provided in the agent's INN record is: all-P-ambo-[2′,3′-Azanediyl-P-(dimethylamino)-P,2′,3′-trideoxy-2′,3′-seco](2′-N→5′)(G-T-T-G-C-C-T-C-C-G-G-T-T-C-T-G-A-A-G-G-T-G-T-T-C) 5′-{P-[4-({2-[2-(2-hydroxyethoxy)ethoxy]ethoxy}carbonyl)piperazin-1-yl]-N,N-dimethylphosphonamidate}, but we have been unable to resolve this to a SMILES or HELM notation. |
| References |
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1. Anwar S, Yokota T. (2020)
Golodirsen for Duchenne muscular dystrophy. Drugs Today (Barc), 56 (8): 491-504. [PMID:33025945] |
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2. Carver MP, Charleston JS, Shanks C, Zhang J, Mense M, Sharma AK, Kaur H, Sazani P. (2016)
Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates. J Neuromuscul Dis, 3 (3): 381-393. [PMID:27854228] |
