| 发明名称 |
RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF DUX4 |
| 摘要 |
The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy. |
| 申请公布号 |
US2017029849(A1) |
申请公布日期 |
2017.02.02 |
| 申请号 |
US201615295591 |
申请日期 |
2016.10.17 |
| 申请人 |
NATIONWIDE CHILDREN'S HOSPITAL, INC. |
发明人 |
Harper Scott Quenton;Liu Jian;Coppens Sara;Wallace Lindsay |
| 分类号 |
C12N15/86;C12N15/113 |
主分类号 |
C12N15/86 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A recombinant adeno-associated virus comprising the DUX4 miRNA-encoding DNA set out in SEQ ID NO: 1 or 2, wherein the recombinant adeno-associated virus lacks rep and cap genes. |
| 地址 |
Columbus OH US |
