发明名称 SiRNA therapy for transthyretin (TTR) related ocular amyloidosis
摘要 The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the retinal pigment epithelium of the subject.
申请公布号 AU2011235276(A1) 申请公布日期 2012.09.20
申请号 AU20110235276 申请日期 2011.03.29
申请人 JONO, HIROFUMI;KUMAMOTO UNIVERSITY;ALNYLAM PHARMACEUTICALS, INC.;ANDO, YUKIO 发明人 ANDO, YUKIO;JONO, HIROFUMI;ALVAREZ, RENE;SAH, DINAH WEN-YEE
分类号 A61K31/7088;C07H21/02 主分类号 A61K31/7088
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