| Description | Inotersen sodium, also known as ISIS-420915 sodium, is a 2′-O-methoxyethyl-modified antisense oligonucleotide compound. It effectively inhibits transthyretin (TTR) protein production by targeting the TTR RNA transcript, thereby reducing TTR transcript levels. Inotersen sodium holds potential for research on hereditary TTR amyloidosis polyneuropathy. |
| In vitro | Inotersen sodium (0.16-20 μM; 16 h) dose-dependently reduces TTR mRNA levels in HepG2 cells[1]. |
| In vivo | 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]. |
| Synonyms | ISIS-420915 sodium |
| molecular weight | N/A |
| CAS | 1432726-13-0 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| References | 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. |