VIRAL GENE THERAPY AS TREATMENT FOR CHOLESTEROL STORAGE DISEASE OR DISORDER
- 专利权人:
- 发明人:
- Charles P. Venditti,Randy Chandler,William J. Pavan
- 申请号：
- US15565065
- 公开号：
- US20180104289A1
- 申请日:
- 2016.04.07
- 申请国别(地区):
- US
- 年份:
- 2018
- 代理人:
- 摘要：
- Provided herein are compositions and methods for the viral gene therapy (e.g., AAV-directed gene therapy) of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.
- 来源网站：
- 中国工程科技知识中心
