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].
Molecular Weight:
8647.00
Purity:
92.00
CAS Number:
[1422959-91-8]
Formula:
C305H481N138O112P25
Target:
Dystrophin
Application Notes:
Oligonucleotides
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