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 Weight:
8647.40
Purity:
92.00
Formula:
C305H481N138Na25O112P25
Target:
DNA/RNA Synthesis,Dystrophin
Application Notes:
Oligonucleotides
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