发明名称 |
COMPOSITIONS AND THEIR USES DIRECTED TO HUNTINGTIN |
摘要 |
Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. |
申请公布号 |
US2016369273(A1) |
申请公布日期 |
2016.12.22 |
申请号 |
US201615142827 |
申请日期 |
2016.04.29 |
申请人 |
Ionis Pharmaceuticals, Inc. |
发明人 |
Freier Susan M. |
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. An antisense oligonucleotide 12 to 35 nucleotides in length comprising at least 12 consecutive nucleotides of a nucleotide sequence selected from the group consisting of SEQ ID NOs 53-65. |
地址 |
Carlsbad CA US |