生物活性: Inotersen (ISIS-420915) sodium is a 2′-O-methoxyethyl-modified antisense oligonucleotide. Inotersen sodium inhibits the production of transthyretin (TTR) protein by targeting the TTR RNA transcript and reduces the levels of the TTR transcript. Inotersen sodium can be used for the research of hereditary TTR amyloidosis polyneuropathy.
体内研究: Inotersen sodium (10-100 mg/kg; s.c. twice a week for 4 weeks) reduces the plasma TTR protein by >80% in transgenic mice bearing the Ile84Ser human TTR mutant.