Methods and Compositions for Gene Delivery
- 专利权人:
- HADASIT MEDICAL RESEARCH SERVICES & DEVELOPMENT LTD.
- 发明人:
- Stella Mitrani-Rosenbaum,Avizohar Argov
- 申请号：
- US14370594
- 公开号：
- US20150045416A1
- 申请日:
- 2013.01.03
- 申请国别(地区):
- US
- 年份:
- 2015
- 代理人:
- 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.
- 来源网站：
- 中国工程科技知识中心
