Golodirsen (sodium)
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].
Product Specifications
Product Name Alternative
SRP-4053 (sodium)
UNSPSC
12352208
Target
DNA/RNA Synthesis; Dystrophin
Type
Oligonucleotides
Related Pathways
Cell Cycle/DNA Damage; Cytoskeleton
Field of Research
Neurological Disease
Assay Protocol
https://www.medchemexpress.com/golodirsen-sodium.html
Purity
92.00
Solubility
10 mM in H2O
Smiles
[Golodirsen(sodium)]
Molecular Formula
C305H481N138Na25O112P25
Molecular Weight
8647 (free acid)
References & Citations
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, stored under nitrogen, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Launched
Available Sizes
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