THE SYDNEY CHILDREN'S HOSIPITALS NETWORKI (RANDWICK AND WESTMEAD);CAMBRIDGE ENTERPRISE LIMITED
发明人:
Peter Lachmann,Ian Alexander
申请号：
US16099880
公开号：
US20200147240A1
申请日:
2017.05.03
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Methods of treatment of complement-mediated disorders, in particular disorders associated with over-activity of the complement C3b feedback cycle (for example, age-related macular degeneration (AMD)), using gene therapy is described. According to the methods, levels of complement Factor I are elevated by administration of a recombinant viral vector encoding Factor I such that a therapeutically effective amount of the encoded Factor I is expressed from the vector in the subject. Recombinant viral vectors encoding Factor I, recombinant virus particles encapsidating the vectors, and their use in the methods of treatment, is also described.
