Golodirsen sodium
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Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD).
For research use only. We do not sell to patients.
- Purity: 92.00%
- Formula: C305H481N138Na25O112P25
- Molecular Weight:8647 (free acid)
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Storage:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
All DNA/RNA Synthesis Isoforms
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Biological Activity
Golodirsen sodium demonstrates efficient and specific exon 53 skipping ability in human fibroblasts, and is able to significantly restore the imbalance of DMD transcripts[1].
Golodirsen sodium restores the expression of the anti-myostatin protein in human fibroblasts, but there are significant individual differences in the level of protein recovery, and it has no correlation with the jumping efficiency[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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Appearance Solid
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Molecular Weight 8647 (free acid)
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Formula C305H481N138Na25O112P25
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Color White to off-white
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SMILES
[Golodirsen (sodium)]
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Synonyms
SRP-4053 sodium
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Purity & Documentation
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Data Sheet (266 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2242 KB)
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)