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) [1][2].
Molecular Formula
C305H481N138Na25O112P25
Smiles
[Golodirsen(sodium)]
Target
DNA/RNA Synthesis; Dystrophin
Purity
92
Solubility
10 mM in H2O
Storage Conditions
-20°C (Powder, stored under nitrogen, away from moisture)
