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

[1]Goey, Andrew KL, et al. Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy. Drug Metabolism and Disposition 52.12 (2024) : 1396-1406.|[2]Rossi R, et al. Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells. Skelet Muscle. 2024 Nov 29;14 (1) :28.