ANNA BORODOVSKY,BRIAN BETTENCOURT,DONALD FOSTER,JAMES BUTLER,KALLANTHOTTATHIL G. RAJEEV,KEVIN FITZGERALD,KLAUS CHARISSE,MARTIN MAIER,MUTHIAH MANOHARAN,SATYANARAYANA KUCHIMANCHI
申请号：
BR112015022876
公开号：
BR112015022876A2
申请日:
2014.03.13
申请国别(地区):
BR
年份:
2017
代理人:
摘要：
complement component c5 irna compositions and methods of use thereof abstract of the invention the invention relates to irna, eg, double-stranded ribonucleic acid (dsrna), compositions targeting the complement component c5 gene, and methods of using such irna, eg, dsrna, compositions to inhibit expression of c5 and to treat subjects having a complement component c5-associated disease, eg, paroxysmal nocturnal hemoglobinuria. 1 the species oligo name shows the genbank record (e.g., nm_001735.2) and the position in the nucleotide sequence of the genbank record (e.g., 1517-1539) that the antisense strand targets. 2 the number following the decimal point refers to the lot number. 3 um = unmodified abstract summary "a double-stranded ribonucleic acid agent (dsrna) and a double-stranded RNA inhibitor of expression of the complement component c5, a double-stranded RNA-inhibiting agent capable of inhibiting expression of the c5 complement component in a cell, a cell, a vector encoding at least one strand of a dsrna agent, a pharmaceutical composition for inhibiting expression of a c5 complement component gene, a method of inhibiting expression of the cs complement component. c5 complement in a cell, a method of treating a subject having a disease or disorder that would benefit from reduced expression of the c5 complement component, and a method of preventing at least one symptom in a subject having a disease or disorder that would benefit from reduction in expression of complement component c5 "the invention relates to irna, e.g., double-stranded ribonucleic acid (dsrna), compositions addressing the c5 complement component gene, and methods of using such a cna, e.g., dsrna, compositions for inhibiting c5 expression and for treating subjects having a c5 complement component-associated disease, e.g. , paroxysmal nocturnal hemoglobinuria. 24/24 1/1complement component c5 irna compositions and methods of use thereof abstract of the invention the invention relates to irna, e.g.,
