golodirsen
Ligand Summary
Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. It is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
ChEMBL: CHEMBL4297762
DrugCentral: 5357
LyCHI: golodirsen
Target Activities
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ANTISENSE INHIBITOR | ||||||