| 发明名称 |
COMPOUNDS FOR TREATING, DELAYING AND/OR PREVENTING A HUMAN GENETIC DISORDER SUCH AS MYOTONIC DYSTROPHY TYPE I (DMI) |
| 摘要 |
The current invention provides new compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1), spino-cerebellar ataxia 8 and/or Huntington's disease-like 2 caused by expansions of CUG repeats in the transcripts of DM1/DMPK, SCA8 or JPH3 genes. |
| 申请公布号 |
US2017029820(A1) |
申请公布日期 |
2017.02.02 |
| 申请号 |
US201615094212 |
申请日期 |
2016.04.08 |
| 申请人 |
BioMarin Technologies B.V. |
发明人 |
Aguilera Diez Maria Begoña;de Visser Peter Christian;Mulders Susan Allegonda Maria |
| 分类号 |
C12N15/113;C07K19/00;A61K31/7088;A61K47/48 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A compound comprising: a peptide having the sequence LGAQSNF (SEQ ID NO: 2) conjugated to a 2′-O-methyl phosphorothioate oligonucleotide having the sequence cagcagcagcagcagcagcag (SEQ ID NO: 1). |
| 地址 |
Leiden NL |
