发明名称 |
GENE THERAPY FOR RETINITIS PIGMENTOSA |
摘要 |
Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated. |
申请公布号 |
CA2943185(A1) |
申请公布日期 |
2015.09.24 |
申请号 |
CA20152943185 |
申请日期 |
2015.03.20 |
申请人 |
GENZYME CORPORATION |
发明人 |
O'RIORDAN, CATHERINE;ADAMOWICZ, MATTHEW |
分类号 |
A61K48/00;A61K31/7105;C12N15/86 |
主分类号 |
A61K48/00 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|