Ligand id: 7364

Name: mipomersen

Structure and Physico-chemical Properties

2D Structure
No information available.
Summary of Clinical Use
Used as an adjunct to diet and other lipid-lowing medications in the treatment of homozygous familial hypercholesterolemia.
Mechanism Of Action and Pharmacodynamic Effects
Mipomersen is a cholesterol-reducing antisense therapeutic. It targets the messenger RNA for apolipoprotein B. Mipomersen binds to the mRNA of one of apolipoprotein B's isoforms, Apo B-100 (Uniprot P04114). The resulting double-stranded RNA is degraded by RNase H, preventing translation of the mRNA and formation of the apo B-100 protein. Mipomersen binds at nucleotide positions 3249-3268 relative to the published sequence provided in GenBank accession number NM_000384.
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