发明名称 |
RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN |
摘要 |
The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A. |
申请公布号 |
WO2012145597(A2) |
申请公布日期 |
2012.10.26 |
申请号 |
WO2012US34408 |
申请日期 |
2012.04.20 |
申请人 |
NATIONWIDE CHILDREN'S HOSPITAL, INC.;HARPER, SCOTT QUENTON;LIU, JIAN |
发明人 |
HARPER, SCOTT QUENTON;LIU, JIAN |
分类号 |
C12N7/01;A61K31/7088;A61K48/00 |
主分类号 |
C12N7/01 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|