| 发明名称 |
ENHANCED AAV-MEDIATED GENE TRANSFER FOR RETINAL THERAPIES |
| 摘要 |
Described herein are capsid proteins and adeno-associated viruses capable of targeting various types of ocular cells including bipolar and horizontal cells. Also described herein are methods of treating various ocular disorders in a subject in need thereof by administering to the subject an effective concentration of a composition comprising the recombinant adeno-associated virus (AAV) of the invention. |
| 申请公布号 |
US2015376240(A1) |
申请公布日期 |
2015.12.31 |
| 申请号 |
US201414766172 |
申请日期 |
2014.02.07 |
| 申请人 |
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA |
发明人 |
Cronin Therese;Bennett Jean;Vandenberghe Luk E. |
| 分类号 |
C07K14/005;C12N15/86;C12N7/00;C07K7/06 |
主分类号 |
C07K14/005 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A recombinant AAV capsid protein characterized by a mutation in aa 587-595 as compared to the wild type AAV8 vp1 capsid sequence, or a mutation in the analogous region of another AAV capsid as compared to the corresponding AAV wild type capsid sequence. |
| 地址 |
Philadelphia PA US |
