Fig. 1 · Structure
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Golodirsen
Antisense Oligonucleotide
Golodirsen is an antisense oligonucleotide designed to target the Duchenne muscular dystrophy (DMD) gene by utilizing a phosphorodiamidate morpholino oligomer (PMO) structure. It functions by modifying the splicing of pre-mRNA to skip exon 53, effectively restoring the reading frame of the DMD gene. Golodirsen can be employed in research focused on DMD, helping to investigate potential therapies aimed at restoring the expression of the anti-myostatin protein and improving muscle function.
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Specifications
| Manufacturer | Adooq Bioscience |
|---|---|
| CAS Number | 1422959-91-8 |
| Applications | Antisense Oligonucleotide |
| Molecular Weight | 8647.00 |
| Synonyms | SRP-4053 |
Storage & Solubility
In Vitro Solubility
Reported Solubility
No reported solubility data are available for this product.
Preparing Stock Solutions
| Concentration | |
|---|---|
Values use four significant digits. Prepare to the final solution volume; this is not the volume of solvent to add.
Stock preparation is unavailable for this product molecular weight.
Calculated stock concentrations do not establish solvent solubility. Refer to the reported solubility data for experimental guidance.
Storage
Store powder at -20°C, keep desiccated.
In powder form, the chemical is stable for 36 months.
In solution, store at -20°C and use within 1 month to prevent loss of potency. Aliquot to avoid multiple freeze/thaw cycles.