Inotersen is a 2'-O-methoxyethyl-modified antisense oligonucleotide targeting transthyretin (TTR) to inhibit its production. By binding to TTR mRNA, Inotersen promotes RNase H1-mediated degradation, leading to a decrease in both mutant and wild-type TTR synthesis in the liver. This reduction significantly mitigates amyloid fiber deposition, making Inotersen a valuable tool in research focused on hereditary transthyretin amyloidosis, including its related complications such as polyneuropathy and cardiomyopathy. Caution is advised, as high doses may lead to specific toxicities, including inflammation or tumor development in certain animal models.
Inotersen is a 2'-O-methoxyethyl-modified antisense oligonucleotide targeting transthyretin (TTR) to inhibit its production. By binding to TTR mRNA, Inotersen promotes RNase H1-mediated degradation, leading to a decrease in both mutant and wild-type TTR synthesis in the liver. This reduction significantly mitigates amyloid fiber deposition, making Inotersen a valuable tool in research focused on hereditary transthyretin amyloidosis, including its related complications such as polyneuropathy and cardiomyopathy. Caution is advised, as high doses may lead to specific toxicities, including inflammation or tumor development in certain animal models.
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