The United States of America; as represented by the Secretary; Department of Health and Human Services
发明人:
申请号：
EP16717228.7
公开号：
EP3280451B8
申请日:
2016.04.07
申请国别(地区):
EP
年份:
2019
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.
