发明名称 |
Organic Compositions to Treat Beta-ENaC-Related Diseases |
摘要 |
The present disclosure relates to RNAi agents useful in methods of treating BetaENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC. |
申请公布号 |
US2016272979(A1) |
申请公布日期 |
2016.09.22 |
申请号 |
US201615166576 |
申请日期 |
2016.05.27 |
申请人 |
Arrowhead Pharmaceuticals, Inc. |
发明人 |
De Fougerolles Antonin;Diener John;Hickman Emma;Hinkle Gregory;Milstein Stuart;Pulichino Anne-Marie;Sprague Andrew Griffin |
分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
1. A composition comprising a RNAi agent comprising a first strand and a second strand, wherein: the sequence of the first strand comprises the sequence of nt 1-19 of any sequence disclosed in Table 1; wherein the length of the first and second strand are each no more than about 30 nucleotides; and wherein the first and/or second strand is modified or unmodified. |
地址 |
Pasadena CA US |