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[1][2][3].
Inotersen sodium (0.16-20 μM; 16 h) dose-dependently reduces TTR mRNA levels in HepG2 cells[1].
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[1].
[1]. Ackermann EJ, et, al. Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides. Amyloid. 2016 Sep;23(3):148-157.
[2]. Mathew V, et, al. Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis. Drug Des Devel Ther. 2019 May 6;13:1515-1525.
[3]. Benson MD, et, al. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):22-31.