1422959-91-8
1422959-91-8
Chemical Structure
Golodirsen
Synonym(s): SRP-4053
- CAS No.: 1422959-91-8
- Formula:N/A
- Molecular Weight:8647.00
SMILES: [Golodirsen]
Biological Activity: Golodirsen (SRP-4053) is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen can restore the expression of the anti-myostatin protein. Golodirsen can be used for the research of duchenne muscular dystrophy (DMD)[1][2].
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Golodirsen | Golodirsen (SRP-4053) is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen can restore the expression of the anti-myostatin protein. Golodirsen can be used for the research of duchenne muscular dystrophy (DMD). | |||||||||||||||||||||
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References
- [1]. Rossi R, et al. Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells. Skelet Muscle. 2024 Nov 29;14(1):28. [Content Brief]
- [2]. Goey, Andrew KL, et al. Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy. Drug Metabolism and Disposition 52.12 (2024): 1396-1406.
Keywords
