Golodirsen

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].

Product Specifications

CAS Number

[1422959-91-8]

Product Name Alternative

SRP-4053

UNSPSC

12352208

Target

Dystrophin

Type

Oligonucleotides

Related Pathways

Cytoskeleton

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/golodirsen.html

Purity

92.00

Solubility

H2O : 100 mg/mL (ultrasonic)

Smiles

[Golodirsen]

Molecular Formula

C305H481N138O112P25

Molecular Weight

8647

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.