| 发明名称 |
Methods and Compositions for Gene Delivery |
| 摘要 |
Disclosed herein are AAV-based viral vectors encoding GNE from muscle-specific and non-muscle specific promoters, and the use of same in treating myopathies associated with altered GNE function. |
| 申请公布号 |
US2015045416(A1) |
申请公布日期 |
2015.02.12 |
| 申请号 |
US201314370594 |
申请日期 |
2013.01.03 |
| 申请人 |
HADASIT MEDICAL RESEARCH SERVICES & DEVELOPMENT LTD. |
发明人 |
Mitrani-Rosenbaum Stella;Argov Avizohar |
| 分类号 |
C12N15/86;A61K45/06;A61K31/713 |
主分类号 |
C12N15/86 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. An adeno-associated virus (AAV)-based viral vector, comprising a nucleotide sequence encoding a UDP-N acetylglucosamine 2 epimerase/N-acetylmannosamine kinase (GNE) functionally linked to a muscle-specific promoter. |
| 地址 |
Jerusalem IL |
