COMPOSITION AND METHOD FOR TREATING COMPLEMENT-MEDIATED DISEASE
A recombinant vector having an expression cassette comprising a modified human factor H (hfH) gene is provided, wherein said hfH gene encodes a hfH protein variant comprising SCR1-4, 19-20, and one or more of SCR7, SCR17 and/or SCR18. Also provided are pharmaceutical compositions containing this vector and uses therefor in treating AMD and/or other complement associated diseases.
1 . A recombinant vector having packaged therein an expression cassette comprising an engineered human complement regulator factor H (hfH) gene operably linked to expression control sequences which direct expression thereof, wherein said hfH gene encodes a soluble hfH protein variant that retains complement regulatory function, wherein said fH variant comprises:
short consensus repeat (SCR) 1, 2, 3, 4, 19, 20 and one or more of SCR7, 17 and/or 18, wherein following administration of the vector to a subject and expression, detectable plasma levels of the hfH variant are present in the subject for at least a week.