Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

被引：59

作者：

Mathew, Veena ^{[1
]}

Wang, Annabel K. ^{[1
,2
]}

机构：

[1] Univ Calif Irvine, Dept Neurol, UCI ALS & Neuromuscular Ctr, Orange, CA 92668 USA

[2] Tibor Rubin VA Med Ctr, Neurol Sect, Long Beach, CA USA

来源：

DRUG DESIGN DEVELOPMENT AND THERAPY | 2019年 / 13卷

关键词：

antisense oligonucleotide; familial amyloid polyneuropathy; Inotersen; mRNA; RNase H; ANTISENSE OLIGONUCLEOTIDE; LIVER-TRANSPLANTATION; MOLECULAR-MECHANISMS; POLYNEUROPATHY; DIFLUNISAL; THERAPY; GENE;

D O I：

10.2147/DDDT.S162913

中图分类号：

R914 [药物化学];

学科分类号：

100701 ;

摘要：

Hereditary transthyretin amyloidosis is a fatal autosomal dominant disorder characterized by deposition of transthyretin amyloid into the peripheral nervous system, heart, kidney, and gastrointestinal tract. Previous treatments using liver transplantation and small molecule stabilizers were not effective in stopping disease progression. Inotersen, a 2'-O-methyoxyethyl-modified antisense oligonucleotide, which acts by reducing the production of transthyretin, was recently demonstrated to improve disease course and quality of life in early hereditary transthyretin amyloidosis polyneuropathy in a 15-month Phase III study.

引用

页码：1515 / 1525

页数：11

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