Inotersen (GSK-2998728, ISIS-420915) is a 2-O-methoxyethyl-modified antisense oligonucleotide and transthyretin (TTR) inhibitor with low genotoxicity. Inotersen triggers RNase H1-mediated degradation by binding to TTR mRNA, thereby effectively reducing the production of both mutant and wild-type transthyretin in the liver. Inotersen significantly reduces amyloid fiber deposition, yet specific toxicities such as inflammation or tumors are observed at high doses in some animal models. Inotersen is used in studies of hereditary transthyretin amyloidosis and the associated polyneuropathy and cardiomyopathy[1][2][3][4].
Molecular Weight:
7183.08
Purity:
96.44
CAS Number:
[1492984-65-2]
Formula:
C230H318N69O121P19S19
Target:
Transthyretin (TTR)
Application Notes:
MCE Product type: Oligonucleotides
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