The Sydney Children's Hospitals Network (Randwick and Westmead);Cambridge Enterprise Limited
发明人:
Lachmann, Peter,Alexander, Ian
申请号：
AU2017264750
公开号：
AU2017264750A1
申请日:
2017.05.03
申请国别(地区):
AU
年份:
2018
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.
