| 发明名称 | Antisense modulation of superoxide dismutase 1, soluble expression | ||
| 摘要 | Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding superoxide dismutase 1, soluble. Methods of using these compounds for modulation of superoxide dismutase 1, soluble expression and for treatment of diseases associated with expression of superoxide dismutase 1, soluble are provided. | ||
| 申请公布号 | US9476051(B2) | 申请公布日期 | 2016.10.25 |
| 申请号 | US201514630581 | 申请日期 | 2015.02.24 |
| 申请人 | Ionis Pharmaceuticals, Inc. | 发明人 | Bennett C. Frank;Dobie Kenneth W.;Freier Susan M.;Condon Thomas P.;Jain Ravi;Smith Richard Alan;Cleveland Don W. |
| 分类号 | A61K48/00;C07H21/02;C07H21/04;C12N15/11;C12N15/113;A61K38/00 | 主分类号 | A61K48/00 |
| 代理机构 | McNeill Baur PLLC | 代理人 | McNeill Baur PLLC |
| 主权项 | 1. A method of treating amyotrophic lateral sclerosis (ALS) comprising administering to the cerebrospinal fluid of a subject in need thereof a therapeutically or prophylactically effective amount of a composition comprising: a modified oligonucleotide consisting of 12 to 30 linked nucleosides targeted to SOD-1, wherein the modified oligonucleotide is specifically hybridizable to the coding region of SOD-1, and a pharmaceutically acceptable diluent or carrier. | ||
| 地址 | Carlsbad CA US | ||