CAS NO: | 1637600-16-8 |
包装 | 价格(元) |
100mg | 电议 |
250mg | 电议 |
500mg | 电议 |
生物活性 | Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3-7a)-conjugated antisense oligonucleotide targetingtransthyretin (TTR)mRNA to inhibit production of both variant and wild-type TTR protein. Misfolded TTR induces amyloid fibrils formation in the heart and peripheral nerves, leads to amyloid TTR (ATTR) amyloidosis diseases[1][2][3]. | ||||||||
IC50& Target | TTR[1], asialoglycoprotein receptor[3] | ||||||||
体外研究 (In Vitro) | Eplontersen mediates N-acetylgalactosamine moiety targeting the oligonucleotide to cells bearing an asialoglycoprotein receptor[3]. | ||||||||
体内研究 (In Vivo) | Eplontersen (682884) (0.6, 2, 6 mg/kg; s.c.; once a week for 3 weeks) inhibits TTR protein expression in a dose-dependent manner in vivo, without affecting normal growth in transgenic C57BL/6 mice expressing human transthyretin (TTR)[3].
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Clinical Trial | |||||||||
CAS 号 | 1637600-16-8 | ||||||||
运输条件 | Room temperature in continental US; may vary elsewhere. | ||||||||
储存方式 | Please store the product under the recommended conditions in the Certificate of Analysis. |