补体组分C5 iRNA组合物及其使用方法
本发明涉及iRNA例如双链核糖核酸(dsRNA)、靶向补体组分C5基因的组合物以及使用此种iRNA(例如dsRNA)、组合物来抑制C5的表达并且治疗患有一种补体组分C5相关联的疾病(例如,阵发性睡眠性血红蛋白尿症)的受试者的方法。 The invention relates to iRNA, e.g. , double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, co...
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Format | Patent |
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Language | Chinese |
Published |
23.11.2021
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Subjects | |
Online Access | Get full text |
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Summary: | 本发明涉及iRNA例如双链核糖核酸(dsRNA)、靶向补体组分C5基因的组合物以及使用此种iRNA(例如dsRNA)、组合物来抑制C5的表达并且治疗患有一种补体组分C5相关联的疾病(例如,阵发性睡眠性血红蛋白尿症)的受试者的方法。
The invention relates to iRNA, e.g. , double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g. , paroxysmal nocturnal hemoglobinuria. |
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Bibliography: | Application Number: CN20148027279 |