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Golodirsen

CAT: 0804-HY-132611-01Size: 1 mgDry Ice: NoHazardous: No
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CAT#:0804-HY-132611-01Size:1 mg
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Description
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].
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.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, stored under nitrogen, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Launched

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CatalogName
1422959-91-8Golodirsen

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