转甲状腺素
淀粉样变性
医学
RNA干扰
内科学
生物信息学
生物
遗传学
基因
核糖核酸
作者
Teresa Coelho,David Adams,Ana Martins da Silva,Pierre Lozeron,Philip N. Hawkins,Timothy Mant,Javier Martínez Pérez,Joseph Chiesa,Steve Warrington,Elizabeth Tranter,Malathy Munisamy,Rick Falzone,Jamie Harrop,Jeffrey Cehelsky,Brian R. Bettencourt,Mary Geissler,James S. Butler,Alfica Sehgal,Rachel Meyers,Qingmin Chen
标识
DOI：10.1056/nejmoa1208760
摘要
Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin.
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