| 发明名称 |
COMPOSITIONS AND METHODS FOR MODULATING DYSFERLIN EXPRESSION |
| 摘要 |
Aspects of the disclosure relate to methods of altering RNA splicing in a subject. In some embodiments, methods are provided for correcting splicing in a cell that contains a DYSF gene having a mutation that results in defective splicing. |
| 申请公布号 |
WO2015187825(A3) |
申请公布日期 |
2016.03.17 |
| 申请号 |
WO2015US33973 |
申请日期 |
2015.06.03 |
| 申请人 |
UNIVERSITY OF MASSACHUSETTS |
发明人 |
BROWN, ROBERT, H., JR.;DOMINOV, JANICE, A. |
| 分类号 |
C12Q1/68;C07K16/18;C12N15/113 |
主分类号 |
C12Q1/68 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
|
| 地址 |
|
